FDA Approves Dawnzera (donidalorsen) as the First and Only RNA-targeted Prophylactic Treatment for Hereditary Angioedema

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced the U.S. Food and Drug Administration (FDA) approval of Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. Dawnzera is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE. Dawnzera 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W).

HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the U.S.

“Dawnzera represents a significant advance for people living with HAE who need improved treatment options. With strong and durable efficacy, convenient administration and the longest dosing option available, we believe Dawnzera will be the prophylactic treatment of choice for many people living with HAE. Importantly, the recently published switch data empowers patients and physicians with a roadmap for switching to Dawnzera from other prophylactic therapies,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “At Ionis, we are dedicated to turning groundbreaking science into life-changing medicines. With the early success of our first independent launch of TRYNGOLZA® for familial chylomicronemia syndrome (FCS), and now with Dawnzera, our second independent medicine approved in less than nine months, we are proudly delivering on that vision. To the patients, families, advocacy partners and investigators who helped make this moment a reality, we express our deepest gratitude.”

The approval of Dawnzera was based on positive results from the Phase 3 global, multicenter, randomized, double-blind, placebo-controlled OASIS-HAE study in patients with HAE. The study met its primary endpoint, with Dawnzera Q4W significantly reducing monthly HAE attack rate by 81% compared to placebo over 24 weeks. Mean attack rate reduction increased to 87% when measured from the second dose, a key secondary endpoint. Additionally, Dawnzera Q4W reduced moderate-to-severe HAE attacks by ~90% over 24 weeks when measured from the second dose.

These results are bolstered by the ongoing OASISplus open-label extension (OLE) study, in which Dawnzera Q8W had a similar effect as Q4W over time. Dawnzera demonstrated 94% total mean attack rate reduction from baseline across both dosing groups after one year in the OLE.

The OASISplus study also includes a switch cohort evaluating Dawnzera Q4W in patients previously treated with lanadelumab, C1-esterase inhibitor or berotralstat for at least 12 weeks. Switching to Dawnzera reduced mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch. A total of 84% of patients surveyed preferred Dawnzera over their prior prophylactic treatment, citing better disease control, less time to administer and less injection site pain or reactions.

Across clinical studies, Dawnzera demonstrated a favorable safety and tolerability profile. The most common adverse reactions (incidence ≥ 5%) were injection site reactions, upper respiratory tract infection, urinary tract infection and abdominal discomfort.

“As the first FDA-approved RNA-targeted therapy for HAE, Dawnzera represents a welcome advance in therapeutic options for preventing attacks. Today’s approval gives people living with HAE and their physicians another important choice for aligning treatment with individual needs,” said Anthony J. Castaldo, CEO & chairman of the board, U.S. Hereditary Angioedema Association (HAEA) and Hereditary Angioedema International (HAEi).

"People living with HAE manage this condition for all their lives, and many continue to face unpredictable, painful and dangerous breakthrough attacks even with current treatments. Durable efficacy is essential in maintaining long-term disease control,” said Marc Riedl, M.D., M.S., clinical director, U.S. HAEA Angioedema Center; University of California, San Diego; OASIS-HAE and OASISplus trial investigator. "Dawnzera is positioned to help meet patient needs, providing substantial and sustained reduction of HAE attacks, continued improvement over time and reduced burden of treatment.”

Dawnzera will be available in the U.S. in the coming days.

Ionis is committed to helping people access the medicines they are prescribed and will offer a suite of services designed to meet the unique needs of the HAE community through Ionis Every Step™. As part of Ionis Every Step, patients and healthcare providers will have access to a wide range of support and resources including dedicated support from a Patient Education Manager, assistance with the insurance approval process, information on affordability programs, access to the Dawnzera Direct digital companion and other ongoing services and resources to help patients stay on track. Visit Dawnzera.com for more information.

https://en.wikipedia.org/wiki/Donidalorsen

FDA Approves Dawnzera (donidalorsen) as the First and Only RNA-targeted Prophylactic Treatment for Hereditary Angioedema

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

In continuation of my update on gemcitabine 

Johnson & Johnson (NYSE:JNJ) announced  the U.S. Food and Drug Administration (FDA) approval of  Inlexzo (gemcitabine intravesical system), a new, potentially practice-changing approach for treating patients with certain types of bladder cancer, addressing the need for additional options following unsuccessful BCG therapy and for patients refusing or ineligible for bladder removal surgery (radical cystectomy). Inlexzo, previously referred to as TAR-200, is indicated for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.1

Inlexzo is designed for patients seeking bladder preservation and is the first and only intravesical drug releasing system (iDRS) to provide extended local delivery of a cancer medication into the bladder. Inlexzo remains in the bladder for three weeks per treatment cycle for up to 14 cycles.1 A healthcare professional places Inlexzo into the bladder using a co-packaged urinary catheter and stylet to insert it into the bladder. Inlexzo is placed in an outpatient setting in a few minutes, without the need for general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.1

“When we acquired this novel therapy in 2019, our ambition was to give patients with bladder cancer a renewed sense of hope and belief,” said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. “In an area that has seen little progress for more than 40 years, Inlexzo delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”

The approval is supported by data from the SunRISe-1 ( NCT04640623) single arm, open-label Phase 2b clinical study.1 Results show 82 percent of patients with BCG-unresponsive NMIBC treated with Inlexzo achieved a complete response (CR), meaning no signs of cancer were found after treatment (95 percent confidence interval [CI], 72, 90).1 This high response rate demonstrated strong durability, and 51 percent of these patients maintained a complete response for at least one year.1

In the SunRISe-1 clinical trial supporting this approval, the most common adverse reactions (≥15 percent) including laboratory abnormalities, were urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased aspartate aminotransferase (AST), decreased sodium, bladder irritation, and increased alanine transaminase (ALT).1

“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved Inlexzo,” said Sia Daneshmand, M.D., TAR-200 SunRISe-1 principal investigator, and Professor of Urology, Director of Urologic Oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California.* “In my experience, Inlexzo is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven’t responded to traditional therapy.”

“We are proud of the science that has brought us to this historic moment,” said John Reed, M.D., Ph.D., Executive Vice President, R&D, Innovative Medicine, Johnson & Johnson. “Inlexzo is a novel therapy with powerful efficacy and demonstrated safety profile. As the only major healthcare company that hosts both pharmaceuticals and medical devices, we leveraged the speed and scale of Johnson & Johnson to accelerate innovation and deliver this important therapy to patients.”

“At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer. This approval represents the kind of progress that brings new options to a community that urgently needs them,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network (BCAN).** “Patients with bladder cancer need guidance and collaboration with providers to navigate bladder-sparing treatment options, including newly approved treatments like Inlexzo, so they can move forward feeling well-informed and confident.”

Leading to today’s approval, the FDA granted Inlexzo Breakthrough Therapy Designation (BTD), Real-Time Oncology Review (RTOR), and Priority Review.

Johnson & Johnson is committed to helping patients access our treatments. Once a patient and their doctor have decided that Inlexzo is right for the patient, J&J withMe provides a simple, comprehensive patient support program offering cost support, a dedicated Care Navigator and educational resources, at no cost to the patient.

About SunRISe-1, Cohort 2SunRISe-1 ( NCT04640623), Cohort 2, was a single arm, open-label Phase 2b clinical study that evaluated the safety and efficacy of Inlexzo monotherapy for BCG-unresponsive NMIBC patients with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for, or elected not to undergo, radical cystectomy. The primary endpoint for Cohort 2 was complete response (CR) rate at any time point, and secondary endpoints include duration of response (DOR).

About Non-Muscle Invasive Bladder Cancer (NMIBC) and the Current Standard of Care Non-muscle invasive bladder cancer (NMIBC) is a type of non-invasive bladder cancer that can be classified as low, intermediate, or high risk depending on the presence of characteristics including tumor size, presence of multiple tumors, and carcinoma in situ (CIS).2 NMIBC with CIS makes up approximately 10 percent of patients with NMIBC.3 The current standard of care for NMIBC is Bacillus Calmette-Guérin (BCG), which is a weakened form of the bacteria found in tuberculosis treatment. Though effective, some patients become unresponsive to it and may experience challenges.4,5 Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy; it is a life-altering surgery with a high degree of morbidity and adverse impact on life, and has a post-surgery mortality rate of three to eight percent.6,7 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.

About Inlexzo

Inlexzo is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. Inlexzo is an intravesical system enabling extended release of gemcitabine into the bladder. It is placed in a few minutes without general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.

The safety and efficacy of Inlexzo is being evaluated in clinical trials in patients with MIBC in SunRISe-4, and NMIBC in SunRISe-1, SunRISe-3, and SunRISe-

https://en.wikipedia.org/wiki/Gemcitabine#:~:text=More%20than%2010%25%20of%20users,back%20pain%2C%20and%20muscle%20pain.

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

FDA Approves Tonmya (cyclobenzaprine hydrochloride sublingual tablets) for the Treatment of Fibromyalgia

Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP),  announced the U.S. Food and Drug Administration (FDA) approval of  Tonmya™ (cyclobenzaprine HCl sublingual tablets) for the treatment of fibromyalgia in adults. Tonmya is a first-in-class, non-opioid, once-daily bedtime analgesic with a unique sublingual (under the tongue) formulation that is designed for rapid absorption into the bloodstream. Tonmya is the first new FDA-approved therapy for the treatment of fibromyalgia in over 15 years.

“The FDA approval of Tonmya as a first-line treatment for fibromyalgia represents a landmark advancement for the millions of people in the U.S. suffering from the debilitating pain this condition causes,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “At Tonix, we recognized the transformative potential of pursuing a new approach with Tonmya for fibromyalgia, a chronic overlapping pain condition (COPC), that has gone without innovation for many years. We are hopeful that effectively treating pain with Tonmya could help improve the lives of people with this chronic syndrome.”

“The chronic pain of fibromyalgia is debilitating to every aspect of a person’s life, including causing sleep disturbance and fatigue, all of which can negatively impact someone’s ability to carry out their daily activities,” said Sharon Waldrop, a person with lived experience and founder of the Fibromyalgia Association. “For over 15 years, this community has been underserved and waiting for new treatment options. This approval is a promising step forward and brings renewed hope to millions.”

The approval incorporated efficacy from two double-blind, randomized, placebo-controlled, Phase 3 clinical trials of nearly 1,000 patients in total that evaluated Tonmya as a bedtime treatment for fibromyalgia. Across both Phase 3 trials, Tonmya significantly reduced daily pain scores compared to placebo at 14 weeks, the primary endpoint. Additionally, a greater percentage of study participants taking Tonmya experienced a clinically meaningful (≥30%) improvement in their pain after three months, compared to placebo.

Across three Phase 3 clinical trials with over 1,400 patients evaluated, Tonmya was generally well tolerated. The most common adverse events (incidence ≥2% and at a higher incidence in Tonmya-treated patients compared to placebo-treated patients) included oral hypoesthesia (numbness in the mouth), oral discomfort, abnormal product taste, somnolence (drowsiness), oral paresthesia (tingling, pricking or burning in the mouth), oral pain, fatigue, dry mouth, and aphthous ulcer (canker sore).

“For many years, rheumatologists like myself and other healthcare professionals have had to manage fibromyalgia with limited options that do not adequately meet treatment needs for the majority of patients,” said Philip Mease, M.D., Director of Rheumatology Research at the Providence Swedish Medical Center and Clinical Professor at the University of Washington School of Medicine. “Tonmya is a novel treatment approach that targets nonrestorative sleep that is characteristic of fibromyalgia and can impact core symptoms, specifically pain.”

The latest Phase 3 trial, RESILIENT, was recently published in Pain Medicine with data on primary and secondary endpoints measuring pain, patient’s global impression of change, patient-reported symptoms and function, sleep disturbance, and fatigue.

“I know firsthand how the chronic pain of fibromyalgia significantly disrupts my patients’ lives.” Andrea L. Chadwick, M.D., MSc, FASA, Anesthesiology, Pain, and Perioperative Medicine at The University of Kansas Health System. “Treatments that are processed through the liver can result in metabolites that could affect a medicine’s efficacy and safety over time. Tonmya is administered sublingually which is designed to reduce pain quickly and durably with a tolerable safety profile.”

Tonix thanks the participants and investigators involved in its fibromyalgia clinical trials, and FDA for its commitment to approving new treatments for this condition.

Tonmya is expected to be available for adult patients in the U.S. with fibromyalgia beginning in the fourth quarter of this year.

https://en.wikipedia.org/wiki/Cyclobenzaprine

FDA Approves Tonmya (cyclobenzaprine hydrochloride sublingual tablets) for the Treatment of Fibromyalgia

FDA Approves Brinsupri (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

Insmed Incorporated (Nasdaq: INSM),  announced  the U.S. Food and Drug Administration (FDA)  approval of  first-in-class Brinsupri (brensocatib 10 mg and 25 mg tablets), an oral, once-daily treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and children 12 years and older. Brinsupri is the first and only FDA-approved treatment for NCFB, giving hundreds of thousands of patients and clinicians across the U.S. an option to manage this chronic and progressive disease that can lead to permanent lung damage and lung function decline.

"This FDA approval represents a potential paradigm shift in how we approach non-cystic fibrosis bronchiectasis," said Doreen Addrizzo-Harris, M.D., FCCP, the Fiona and Stanley Druckenmiller Professor of Pulmonary, Critical Care and Sleep Medicine at NYU Grossman School of Medicine and Director of the NYU Langone Health Bronchiectasis and NTM Program, and ASPEN investigator. "For the first time, we have a treatment that directly targets neutrophilic inflammation and addresses a root cause of bronchiectasis exacerbations. Based on the strength of the data and the impact we've seen in patients, I believe this could become the new standard in non-cystic fibrosis bronchiectasis care."

There are approximately 500,000 people in the U.S. diagnosed with NCFB, and it is estimated that millions more are living with this disease globally. Unlike other respiratory diseases that are characterized by airway narrowing, bronchiectasis causes airways to permanently widen, making it harder to clear mucus and bacteria, leading to persistent inflammation and infection. A hallmark of bronchiectasis is frequent exacerbations, or flares, when symptoms worsen, such as coughing, increased mucus, shortness of breath and fatigue.

"Non-cystic fibrosis bronchiectasis deeply affects the lives of people living with this chronic lung condition, impacting both their physical health and emotional well-being," added Elisha Malanga, Executive Director of the Bronchiectasis and NTM Association. "Many patients experience frequent flares, which can disrupt daily life and potentially lead to disease progression. The FDA approval of brensocatib represents a significant and long-awaited advancement as the first approved therapy for non-cystic fibrosis bronchiectasis. Our hope is that treatments like this will enable people with bronchiectasis to manage their condition."

This approval is based on data from the Phase 3 ASPEN and Phase 2 WILLOW studies, which were both published in the New England Journal of Medicine. In ASPEN, patients taking Brinsupri 10 mg or 25 mg had a 21.1% and 19.4% reduction in annual rate of exacerbations respectively, as compared to placebo. Both dosage strengths of Brinsupri also met several exacerbation-related secondary endpoints, including significantly prolonging the time to first exacerbation and significantly increasing the proportion of patients remaining exacerbation-free over the treatment period. Patients who received Brinsupri 25 mg experienced statistically significant less decline in lung function, as measured by forced expiratory volume in one second (FEV₁) after using a bronchodilator, at week 52. The safety of Brinsupri was also evaluated in both studies. The most common adverse reactions ≥2% in the ASPEN trial included upper respiratory tract infection, headache, rash, dry skin, hyperkeratosis, and hypertension. The safety profile for adult patients with NCFB in WILLOW was generally similar to ASPEN, except for a higher incidence of gingival and periodontal adverse reactions in WILLOW.

"The FDA approval of the first-ever treatment for non-cystic fibrosis bronchiectasis is a historic milestone for patients and for Insmed," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "By keeping patients at the center of everything we do, we have once again delivered a first-in-class medicine for a disease with no prior approved treatments. This is an incredible achievement in medicine. We're deeply grateful to the patients, providers, and advocates who made this possible – this is just the beginning of what we can accomplish together for this community."

Brinsupri is a first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor, designed to inhibit the activation of enzymes (neutrophil serine proteases) in neutrophils that are key drivers of chronic airway inflammation in NCFB. Brinsupri is the first approved therapy to address the underlying inflammatory process of NCFB.

In parallel, applications for brensocatib with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) have been accepted, and the Company plans to file in Japan in 2025. Commercial launches are anticipated in 2026, pending approval in each territory.

Brinsupri is now available in the U.S. by prescription through a comprehensive specialty pharmacy network.

https://en.wikipedia.org/wiki/Brensocatib

FDA Approves Brinsupri (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

FDA Approves KETARx (ketamine) for Surgical Pain Management

PharmaTher Holdings Ltd. announced     the U.S. Food and Drug Administration (FDA) approval of  KETARx™, on August 8th, 2025, for its indicated uses in surgical pain management. This FDA approval signifies a momentous achievement for Pharma. 

Fabio Chianelli, Founder, Chairman and CEO of PharmaTher, commented:

“Today marks a new chapter for PharmaTher. With FDA approval for ketamine now in hand, we are closer to realizing our goal of becoming a global leader in ketamine-based pharmaceuticals. This historic FDA approval for PharmaTher is a testament to years of dedicated development, signalling a new era of growth. We remain steadfast in our mission to harness the pharmaceutical potential of ketamine for a range of mental health, neurological, and pain disorders.”

Ketamine stands out among psychedelic and psychedelic-adjacent drugs as the only one included on the World Health Organization's Model List of Essential Medicines1. The global ketamine market is expected to experience substantial growth, currently valued at $750 million and projected to reach $3.42 billion by 2034, indicating a compound annual growth rate of 16.4%2. Furthermore, SPRAVATO® (esketamine), an FDA approved treatment for depression, is tracking a sales run rate of $1.6 billion, with guidance anticipating $3 billion to $3.5 billion by 2027-20283. This promising market outlook underscores the potential of KETARx™ and PharmaTher’s strategic position in the industry.

The FDA's approval of the Company’s ketamine product, KETARx™, provides a strong foundation for expanding the development of ketamine across diverse therapeutic areas within the Company's product pipeline. These areas include mental health conditions like depression, neurological disorders such as Parkinson's disease and Amyotrophic Lateral Sclerosis (ALS), and the management of rare or chronic pain, including Complex Regional Pain Syndrome (CRPS).

Since February 2018, ketamine has been regularly listed on the FDA drug shortage list, highlighting a significant need for a consistent, high-quality supply. This issue is cautioned by a compounding risk alert issued by the FDA on October 10, 20234, which detailed potential risks associated with compounded ketamine products used for psychiatric disorders. Robert F. Kennedy Jr., Secretary of Health and Human Services, has also emphasized the importance of expanding research and ensuring legal access to psychedelic therapies for veterans5. The Veterans Health Administration, the largest integrated healthcare system in the U.S., currently approves and funds ketamine infusions for retired military personnel afflicted with depression, PTSD, and chronic pain6. Furthermore, FDA Commissioner Marty Makary's establishment of the Commissioner's National Priority Voucher program signifies the FDA's dedication to expediting access to safe and effective treatments, instilling confidence in potential future regulatory support and accessibility for ketamine.

https://en.wikipedia.org/wiki/Ketamine

FDA Approves KETARx (ketamine) for Surgical Pain Management

FDA Grants Accelerated Approval to Hernexeos (zongertinib) for Previously Treated Patients with HER2-Mutant Advanced NSCLC

Boehringer Ingelheim’s Hernexeos (zongertinib tablets) has been approved by the U.S. Food and Drug Administration (FDA). The kinase inhibitor is indicated for the treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy.1

This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.1

“With the approval of zongertinib, we have an effective, targeted, orally administered treatment option for patients with HER2 (ERBB2)-mutant non-small cell lung cancer in the U.S. that not only elicits a durable response but, importantly, has a manageable safety profile,” said Dr. John Heymach, MD, PhD, chair of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center, and coordinating investigator for the Beamion-LUNG 1 trial. “In a patient population where there are currently limited treatment options, this approval represents a significant advancement in cancer care.”

Accelerated approval is based on data from the Phase Ib Beamion-LUNG 1 trial, demonstrating an objective response rate of 75% (N=71), 6% of patients had a complete response and 69% of patients had a partial response and a duration of response of ≥6 months in 58% of patients (n=53).1 Positive results from the Beamion-LUNG 1 trial were previously presented at the American Association for Cancer Research (AACR) Annual Meeting 2025 and simultaneously published in The New England Journal of Medicine.2

Zongertinib demonstrated a manageable safety profile with a 2.9% discontinuation rate. In the pooled safety population, the most common (> 20%) adverse reactions were diarrhea (53%), hepatotoxicity (27%), rash (27%), fatigue (22%), and nausea (21%).1

“We are grateful to be able to bring forward Hernexeos, which has the potential to reset the benchmark for those living with HER2-mutant advanced non-small cell lung cancer, a condition associated with a particularly poor prognosis,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “Believing in the power of scientific innovation, we aim to provide meaningful improvements to this patient population. Recognizing its potential, we accelerated development to deliver this new treatment option to patients within four years of starting the first clinical trial."

Harnessing the power of precision medicine by targeting HER2 mutations in lung cancer

HER2 (ERBB2) mutations occur in approximately 2–4% of NSCLC cases and are associated with a poor prognosis and higher incidence of brain metastases.3,4,5Alterations in the HER2 (ERBB2) gene, including mutations, amplification and overexpression, trigger uncontrolled cell proliferation, inhibiting cell death, and promoting tumor growth and spread.3,5 Comprehensive biomarker testing using next generation sequencing determines a patient’s eligibility for treatment with zongertinib by identifying HER2 (ERBB2)-mutant advanced NSCLC.1,5

“The advocacy community is thrilled about the approval of zongertinib, as it is another testament to the importance of personalized options for lung cancer that allow for a much more targeted approach for a subgroup of patients who have been waiting many years for innovative treatments,” said Marcia Horn, President and CEO, International Cancer Advocacy Network and Executive Director of the Exon 20 Group/HER2 Warriors. “Understanding your cancer’s unique biomarkers, including HER2, through comprehensive testing is critical for all patients with NSCLC, as it can unlock targeted treatment options.”

About non-small cell lung cancer (NSCLC)

Lung cancer claims more lives than any other cancer type and the incidence is set to increase to over 3 million cases worldwide by 2040.5,6 NSCLC is the most common type of lung cancer.3 Due to a lack of symptoms and misdiagnoses,7 most patients with NSCLC present at stage III or IV, where the disease has metastasized locally or to other organs.8 The estimated 5-year survival rate historically has been less than 10% for metastatic disease.9,10,11 People living with advanced NSCLC can experience a detrimental physical, psychological, and emotional impact on their daily lives.12,13,14

About zongertinib

Zongertinib is a tyrosine kinase inhibitor (TKI) that selectively inhibits HER2 (ERBB2).1 This orally administered, targeted therapy was approved as Hernexeos® (zongertinib tablets) under the FDA’s Accelerated Approval Program, after securing Priority Review as well as Breakthrough Therapy and Fast Track Designations.1

The treatment is being evaluated in ongoing trials, across a range of advanced solid tumors with HER2 alterations.

https://en.wikipedia.org/wiki/Zongertinib#:~:text=Society%20and%20culture-,Legal%20status,under%20the%20brand%20name%20Hernexeos.

FDA Grants Accelerated Approval to Hernexeos (zongertinib) for Previously Treated Patients with HER2-Mutant Advanced NSCLC

FDA Grants Accelerated Approval to Modeyso (dordaviprone) for the Treatment of Recurrent H3 K27M-Mutant Diffuse Midline Glioma

Jazz Pharmaceuticals plc (Nasdaq: JAZZ)  announced the U.S. Food and Drug Administration (FDA) accelerated approval of  Modeyso (dordaviprone) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.1 Continued approval for this indication may be contingent upon verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial.1

Modeyso is the first and only treatment option approved by the FDA for this ultra-rare and aggressive brain tumor that affects an estimated 2,000 people in the U.S. each year, many of whom are children and young adults.2 The disease is characterized by rapid progression and historically has had no effective systemic treatment options.3 To address this urgent unmet patient need, Modeyso is expected to be commercially available in the coming weeks.

"This is a major turning point in neuro-oncology," said Patrick Wen, M.D., Director, Center for Neuro-Oncology, Dana-Farber Cancer Institute and Professor of Neurology, Harvard Medical School. "For the first time, we have an FDA-approved therapy for patients with recurrent H3 K27M-mutant diffuse midline glioma. While outcomes remain challenging for many patients, the objective responses observed with dordaviprone, including durable benefit in some patients, represent a meaningful advancement. This therapy was developed with the underlying biology of the tumor in mind and introduces a new treatment option for a population with historically limited choices."

Modeyso is administered as an oral capsule once weekly. The FDA's decision was based on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma, selected from five open-label clinical studies based on prespecified eligibility criteria. The overall response rate (ORR), as assessed by blinded independent central review (BICR) using Response Assessment in Neuro-Oncology (RANO) 2.0 criteria, was 22% (95% CI: 12 to 36), with an additional responder identified by integrated RANO 2.0. Among responders, the median duration of response was 10.3 months (95% CI: 7.3 to 15.2), with 73% maintaining their response for at least six months and 27% for at least 12 months.1

"The FDA approval of Modeyso is a milestone moment for the patients and families who have long needed new options, the clinicians who have tirelessly searched for solutions, and the researchers and advocates who never gave up," said Joshua E. Allen, Ph.D., Chief Scientific Officer, Chimerix, a Jazz Pharmaceuticals Company. "We're proud to deliver precisely the kind of transformative innovation we strive for, and we congratulate our combined Chimerix and Jazz team, and the community who worked together tirelessly to bring this treatment forward. This approval not only equips clinicians with the first targeted option for this disease but also signals a meaningful shift in what patients and families can expect after diagnosis. We would like to extend our thanks to the patients, advocates, clinicians, principal investigators, scientists, regulators and partner institutions who made this possible."

"This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma," said David F. Arons, President and Chief Executive Officer of the National Brain Tumor Society. "This is a fast-moving, devastating disease that turns families' lives upside down. For years, this diagnosis has lacked an approved treatment and today, that changes. Families finally have a treatment option, and a reason to believe in more time together to make memories that might not have otherwise been possible."

The safety of Modeyso was evaluated in 376 adult and pediatric patients with glioma across four open-label clinical studies. Serious adverse reactions occurred in 33% of patients. Serious adverse reactions reported in more than 2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%) and muscular weakness (2.1%). The most common adverse reactions in patients who received Modeyso (≥20%) were fatigue, headache, vomiting, nausea and musculoskeletal pain.1 See additional safety information below.

About the Phase 2 Clinical Trial Program

The efficacy and safety of Modeyso were evaluated in adult and pediatric patients with glioma across five open-label, non-randomized clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). A pre-specified integrated efficacy analysis included 50 patients with recurrent H3 K27M-mutant diffuse midline glioma who had measurable disease per Response Assessment in Neuro-Oncology (RANO) criteria. Tumor response was assessed every eight weeks by blinded independent central review (BICR). The primary efficacy endpoint was objective response rate (ORR). Safety was evaluated across four of the clinical studies.1

About H3 K27M-Mutant Diffuse Midline Glioma

H3 K27M-mutant diffuse midline glioma is a rare and highly aggressive brain tumor that primarily affects the midline structures of the brain and spinal cord.4,5 It is characterized by a specific genetic mutation (H3 K27M) that disrupts epigenetic regulation and drives tumor growth.6 Most commonly diagnosed in children and young adults, patients with this type of glioma often face an extremely poor prognosis, with limited therapeutic options and very low survival rates following recurrence.7 Median survival is approximately one year from diagnosis and less than six months after progressing following frontline therapy.7

About Modeyso (dordaviprone)

Modeyso (dordaviprone) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.1 Modeyso is an orally administered small molecule given once weekly. Modeyso is a protease activator of the mitochondrial caseinolytic protease P (ClpP) and also inhibits dopamine D2 receptor (DRD2). In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma.1

Modeyso received accelerated approval based on a pre-specified integrated efficacy analysis of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). Continued approval may be contingent upon verification and description of clinical benefit in the ongoing Phase 3 ACTION trial (NCT05580562), which is evaluating the safety and clinical benefit of Modeyso in newly diagnosed patients with H3 K27M-mutant diffuse glioma following radiotherapy.6 Modeyso was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.

Modeyso (dordaviprone) is not approved anywhere else in the world.

https://en.wikipedia.org/wiki/Dordaviprone

FDA Grants Accelerated Approval to Modeyso (dordaviprone) for the Treatment of Recurrent H3 K27M-Mutant Diffuse Midline Glioma

FDA Approves Vostally (ramipril) Oral Solution for the Treatment of Hypertension in Adults

Rosemont Pharmaceuticals, Inc. announced  the U.S. Food and Drug Administration (FDA)  approval of  Vostally (ramipril) Oral Solution, an angiotensin converting enzyme (ACE) inhibitor that offers once-daily dosing in an oral liquid form for patients who have difficulty swallowing.

Vostally is indicated for the treatment of hypertension in adults, to lower blood pressure. Lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions. Additionally, in patients 55 years or older at high risk of developing a major cardiovascular event, Vostally is indicated to reduce the risk of myocardial infarction, stroke, or death from cardiovascular causes. Also, in adult patients with post-myocardial infarction heart failure, Vostally is indicated to reduce the risk of cardiovascular death and hospitalization for heart failure.

Vostally is contraindicated in patients with a history of angioedema or hypersensitivity to this product or any other ACE inhibitor and in patients with hereditary or idiopathic angioedema. Vostally is contraindicated in combination with a neprilysin inhibitor (e.g., sacubitril). Do not administer Vostally within 36 hours of switching to or from sacubitril/valsartan, a neprilysin inhibitor. Do not co-administer Vostally with aliskiren in patients with diabetes.

“We are proud to bring Vostally to physicians to treat patients who may benefit from an ACE inhibitor but whose difficulty swallowing a tablet creates a barrier to this kind of therapy,” said Jeff Bryant, President of Sabal Therapeutics, a Rosemont Company. “This oral liquid form of ramipril provides physicians with another option for treating patients.”

“This FDA approval of Vostally marks an important milestone in expanding treatment options for patients who have difficulty swallowing traditional tablets or capsules,” continued Mr. Bryant. “We are proud to support Rosemont’s commitment to delivering high-quality oral liquid medicines to the U.S. market and believe Vostally will provide a meaningful benefit to both patients and healthcare providers and payors.”

Vostally will be available later this year. Full Prescribing Information, including boxed warning and safety profile, is available at www.RosemontPharmaceuticals.com.

https://en.wikipedia.org/wiki/Ramipril

FDA Approves Vostally (ramipril) Oral Solution for the Treatment of Hypertension in Adults

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced   U.S. Food and Drug Administration (FDA)  approval of Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU.

"We are excited to have reached this important milestone for those affected by PKU," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care. Our experienced customer facing teams are ready to bring this therapy to children and adults with PKU in the United States as quickly as possible."

The FDA approval is based on the evidence of significant efficacy and safety from the

Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.

"The approval marks an exciting milestone for the PKU community," said Catherine Warren, Executive Director of the National PKU Alliance. "This progress brings renewed hope, and we are eager to see the positive impact this new treatment option will have on advancing care and potentially improving quality of life for individuals of all ages and PKU subtypes that respond to this therapy."

Sephience was recently granted marketing authorization by the European Commission. Review of approval applications is ongoing in several other countries including Japan and Brazil.

About Sephience (sepiapterin)

Sephience is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Economic Area and the United States.

Indication and Important Safety Information

Indication

Sephience is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). Sephience is to be used in conjunction with a phenylalanine (Phe)-restricted diet.

Contraindications

None

Important Safety Information

Treatment with Sephience should be directed by physicians knowledgeable in the management of PKU. Biochemical response to Sephience can only be determined by a therapeutic trial with careful monitoring of ongoing dietary and nutritional balance to ensure adequate Phe control.

Warnings and Precautions

Increased Bleeding: Sephience may increase the risk of bleeding. Bleeding events, including superficial hematomas, prolonged bleeding, and heavy menstrual bleeding have occurred in patients treated with Sephience. Inform patients about the risk of bleeding associated with Sephience and have patients follow up with their healthcare provider should such a bleeding event occur. Consider treatment interruption with Sephience in patients with active bleeding.

Hypophenylalaninemia: Some pediatric patients receiving Sephience experienced hypophenylalaninemia. Monitor blood Phe levels during treatment and modify the dosage of Sephience and/or dietary protein and Phe intake as needed to ensure adequate blood Phe level control. Frequent blood monitoring is recommended in the pediatric population.

Interaction with Levodopa: In a 10-year post-marketing safety surveillance program for a non-PKU indication using another drug that is a phenylalanine hydroxylase (PAH) activator, 3 patients with underlying neurological disorders experienced seizures, exacerbation of seizures, over-stimulation, and irritability during co-administration with levodopa. Monitor patients who are receiving levodopa for changes in neurological status during treatment with Sephience.

Adverse Reactions

Most common adverse reactions with Sephience (≥2% and > placebo) were diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain.

Drug Interactions

Avoid concomitant use of drugs known to inhibit folate synthesis dihydrofolate reductase (DHFR) (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, and piritrexim) while taking Sephience. Concomitant administration of such drugs may reduce sepiapterin metabolism to BH4. If concomitant use is not avoidable, monitor blood Phe levels.

Sephience and PDE-5 inhibitors (e.g., sildenafil, vardenafil, or tadalafil) induce vasorelaxation and may reduce blood pressure. Monitor for signs and symptoms of hypotension.

For medical information, product complaints, or to report an adverse event, please call 1–866–562–4620 or email at usmedinfo@ptcbio.com.

https://en.wikipedia.org/wiki/Sepiapterin

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria

FDA Approves Enbumyst (bumetanide) Nasal Spray for the Treatment of Edema Associated with Congestive Heart Failure, Liver Disease and Kidney Disease

Corstasis Therapeutics Inc., announced the U.S. Food and Drug Administration (FDA)  approval of  Enbumyst (bumetanide nasal spray). Enbumyst is indicated for the treatment of edema associated with congestive heart failure (CHF), and hepatic and renal disease, including nephrotic syndrome in adults.

Addressing a Critical Unmet Need

Edema and fluid overload remain the leading causes of hospitalization and readmission for patients with CHF, cirrhosis, and chronic kidney disease. An estimated 6.7 million Americans live with heart failure, and fluid overload drives over 1 million hospitalizations annually, accounting for billions of dollars in U.S. health care expenditures. Oral loop diuretics can be limited by poor gastrointestinal absorption and delayed onset, while intravenous (IV) therapy requires a hospital or infusion setting, often associated with higher resource utilization and increased healthcare expenditure.

Enbumyst demonstrated rapid absorption and predictable diuretic response in clinical studies, with a similar effect on diuresis, natriuresis and urinary potassium excretion when compared to IV bumetanide injection. Enbumyst adds a new self-administered outpatient diuretic therapy, which may help bridge the gap between oral and IV diuretics for treating edema in patients with CHF, liver disease and kidney disease. The FDA-approved Prescribing Information for Enbumyst does not include a boxed warning. Please refer to the Important Safety Information below.

“The FDA approval of Enbumyst represents a meaningful advancement in the treatment of edema for patients and providers,” said Ben Esque, Chief Executive Officer of Corstasis Therapeutics.

“Enbumyst was designed in direct collaboration with cardiologists to address practical challenges in the outpatient care setting,” said Dr. Brian Kolski Chief Medical Director of Corstasis and Director, Non-Invasive Vascular Lab at the Orange County Heart Institute and Director of Structural Heart Disease at Providence St Joseph Hospital in Orange, California.

Commercial Strategy and Market Opportunity

Corstasis expects to launch Enbumyst in the U.S. in the fourth quarter of 2025, targeting cardiologists, nephrologists, hepatologists, outpatient heart failure clinics and integrated delivery networks (IDNs). The company is also preparing a robust market access strategy, including real-world outcomes data and payer partnerships, to support rapid adoption and coverage.

The U.S. market for outpatient diuretic therapies addressing edema associated with CHF, and hepatic and renal disease is estimated to represent a multi-billion-dollar opportunity annually, driven by the high prevalence of heart failure and chronic kidney disease, as well as the unmet need for therapies that reduce costly hospitalizations and readmissions.

“Enbumyst offers the potential to change the standard of care by enabling earlier, outpatient intervention,” said Dr. Anuradha Lala-Trindade, Director of Heart Failure Research at the Mount Sinai Fuster Heart Hospital in New York. “This innovation may meaningfully improve outcomes while potentially easing the economic burden on the healthcare system.”

About Enbumyst

Enbumyst is a nasal spray loop diuretic indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION

Enbumyst is indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

IMPORTANT SAFETY INFORMATION

Enbumyst is contraindicated in patients with anuria, who are in hepatic coma and have a history of hypersensitivity to bumetanide.

Enbumyst is a diuretic that may cause fluid, electrolyte, and metabolic abnormalities. Excessive fluid loss can lead to dehydration, decreased blood volume, and increased risk of blood clots. Abnormalities may include changes in blood electrolytes, nitrogen, glucose, and uric acid. The chance of getting these abnormalities is higher in people who are elderly, use higher doses or who do not get enough electrolytes by mouth.

If increasing azotemia and oliguria occur during treatment of severe progressive renal disease, discontinue bumetanide.

Although unlikely at the recommended doses, the potential for ototoxicity must be considered a risk of intravenous therapy, at high doses, repeated frequently in the face of renal excretory function impairment.

Avoid use in patients with significant nasal mucosal or structural abnormalities, such as acute episodes of rhinitis or congestion due to any cause.

Advise lactating women treated with Enbumyst to monitor their infants for excessive urine output, dehydration, and lethargy.

Ref: https://en.wikipedia.org/wiki/Bumetanide

FDA Approves Enbumyst (bumetanide) Nasal Spray for the Treatment of Edema Associated with Congestive Heart Failure, Liver Disease and Kidney Disease

FDA Approves Harliku (nitisinone) for the Treatment of Patients with Alkaptonuria

In continuation of my update on Nitisinone

Cycle Pharmaceuticals announced the FDA approval of  Harliku (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU.1

Launching in July 2025, Harliku will be the first and only FDA-approved treatment for AKU,1 an ultra-rare genetic metabolic disorder in which patients have a buildup of HGA that leads to osteoarthritis, ochronosis, and complications in the kidneys, and heart.2 Patients with AKU often develop pain, reduced joint mobility, and require large joint replacements; the symptoms impede their physical functionality, emotional well-being, and quality of life.2,3

The approval of Harliku is based on data from a randomized, no-treatment controlled study of 40 patients with AKU. As part of the intramural research program of the National Human Genome Research Institute at the National Institutes of Health (NIH), Dr. Wendy J. Introne, MD and her team showed that nitisinone helped patients improve pain, energy levels, and physical functioning after three years of treatment, assessed using the 36-Item Short-Form Survey.4

Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals commented,

“We are deeply grateful for Cycle’s collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH, whose pioneering work laid the foundation for this FDA approval. We look forward to making Harliku available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities.”

“The approval of Harliku is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU,” said Dr. Wendy J. Introne, MD, of NIH’s National Human Genome Research Institute (NHGRI).

Building on the company’s previous success in rare diseases, Harliku will be Cycle Pharmaceuticals’ eighth commercial product in the US.

Indications

Harliku™ is indicated for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU).

Important Safety Information

Do not prescribe Harliku to patients allergic to nitisinone or any other contained ingredients.

Warnings and Precautions:

Ocular Symptoms and Hyperkeratotic Plaques Due to Elevated Plasma Tyrosine Level:

Ocular signs and symptoms including keratitis, corneal opacities, corneal irritation, corneal ulcers, conjunctivitis, eye pain, and photophobia, have been reported in patients.

Perform slit-lamp examination prior to treatment and regularly thereafter. Reexamine patients if symptoms develop or tyrosine levels are > 500 micromole/L. Assess plasma tyrosine levels in patients with an abrupt change in neurological status.

Perform a clinical laboratory assessment, including plasma tyrosine levels, in patients with an abrupt change in neurological status.

Leukopenia and Severe Thrombocytopenia

In clinical trials, patients with hereditary tyrosinemia type 1 (HT-1) treated with another oral formulation of nitisinone and dietary restriction, developed reversible leukopenia, thrombocytopenia, or both. No patients developed infections or bleeding as a result of the episodes of leukopenia and thrombocytopenia. Monitor platelet and white blood cell counts during Harliku therapy.

Adverse Reactions:

The most common adverse reactions (≥1%) reported in patients with AKU taking nitisinone in clinical trials are elevated tyrosine levels, thrombocytopenia and keratitis.

Drug Interactions:

Nitisinone is a moderate CYP2C9 inhibitor and a weak CYP2E1 inducer. Potential clinical impact of Harliku administration with CYP2C9 substrates. Reduce the dosage of the co-administered drugs metabolized by CYP2C9 by half. Additional dosage adjustments may be needed.

Nitisinone is an inhibitor of OAT1/OAT3. Potential clinical impact of administration with OAT1/OAT3 substrates. Patients should be monitored for potential adverse reactions.

Use in Specific Populations:

Pregnancy: Limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.

Lactation: The developmental and health benefits of breastfeeding should be considered against the mother’s clinical need for Harliku, along with potential adverse effects on the breastfed infant from Harliku or from the underlying maternal condition.

Pediatric Use: The safety and effectiveness of Harliku have not been established in pediatric patients with AKU.

Geriatric Use: Insufficient data from clinical studies of Harliku to determine if patients ≥ 65 years of age respond differently. Elderly patients should be cautious reflecting any decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy.

https://en.wikipedia.org/wiki/Nitisinone

FDA Approves Harliku (nitisinone) for the Treatment of Patients with Alkaptonuria

FDA Approves Yeztugo (lenacapavir) as the First and Only HIV Prevention Option Offering 6 Months of Protection

In continuation of my update lenacapavir

Gilead Sciences, Inc. (Nasdaq: GILD)  announced  the U.S. Food and Drug Administration (FDA)  approval of Yeztugo (lenacapavir)—the company’s injectable HIV-1 capsid inhibitor—as pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35kg, making it the first and only twice-yearly option available in the United States for people who need or want PrEP. Data show that ≥99.9% of participants who received Yeztugo in the Phase 3 PURPOSE 1 and PURPOSE 2 trials remained HIV negative.

“This is a historic day in the decades-long fight against HIV. Yeztugo is one of the most important scientific breakthroughs of our time and offers a very real opportunity to help end the HIV epidemic,” said Daniel O’Day, Chairman and Chief Executive Officer of Gilead Sciences. “This is a medicine that only needs to be given twice a year and has shown remarkable outcomes in clinical studies, which means it could transform HIV prevention. Gilead scientists have made it their life’s work to end HIV and now, with the FDA approval of Yeztugo and in collaboration with our many partners, we can help to make that goal a reality.”

The first PrEP medication, which was also developed by Gilead, was approved in the U.S. in 2012. However, data from the Centers for Disease Control and Prevention (CDC) show that, in 2022 (the most recent year with available data), only about 1 in 3 (36%) people in the U.S. who met the CDC’s eligibility criteria for PrEP were prescribed a form of PrEP. CDC data show that all populations in the U.S. are not yet using PrEP at rates that could end transmission of the virus at the population level, with particular gaps for women, Black/African American and Hispanic/Latino people, and people in the U.S. South. Data also show that barriers including adherence challenges, stigma and low awareness of existing PrEP options—by both healthcare providers and consumers—contribute to this low uptake of PrEP across multiple populations. The potential impact of this limited uptake, adherence and access is underscored by the fact that, in 2023, more than 100 people were diagnosed with HIV every day in the U.S.

“Yeztugo could be the transformative PrEP option we’ve been waiting for—offering the potential to boost PrEP uptake and persistence and adding a powerful new tool in our mission to end the HIV epidemic,” said Carlos del Rio, MD, Distinguished Professor of Medicine in the Division of Infectious Diseases at Emory University School of Medicine and Co-Director of the Emory Center for AIDS Research in Atlanta. “A twice-yearly injection could greatly address key barriers like adherence and stigma, which individuals on more frequent PrEP dosing regimens, especially daily oral PrEP, can face. We also know that, in research, many people who need or want PrEP preferred less frequent dosing.”

FDA approval of Yeztugo is supported by high efficacy and demonstrated safety data in two clinical trials

The FDA approval of Gilead’s New Drug Applications (NDAs) for Yeztugo was supported by data from the Phase 3 PURPOSE 1 and PURPOSE 2 trials conducted by Gilead. In the PURPOSE 1 trial (NCT04994509), data at the primary analysis showed twice-yearly subcutaneous Yeztugo demonstrated zero HIV infections among 2,134 participants in the Yeztugo group, 100% reduction in HIV infections and superiority of prevention of HIV infections when compared with once-daily oral Truvada® (emtricitabine 200mg and tenofovir disoproxil fumarate 300mg; F/TDF) in cisgender women in sub-Saharan Africa. In the PURPOSE 2 trial (NCT04925752), there were two HIV infections among 2,179 participants in the twice-yearly subcutaneous Yeztugo group, demonstrating 99.9% of participants in the Yeztugo group did not acquire HIV infection and superiority of prevention of HIV infections when compared with once-daily oral Truvada among a broad and geographically diverse range of cisgender men and gender-diverse people. In both trials, Yeztugo also demonstrated superiority of prevention of HIV infections when compared with background HIV incidence (bHIV) and was generally well-tolerated, with no significant or new safety concerns identified. Data from both trials were published in The New England Journal of Medicine and, based in part on the trial results, in December 2024 the journal Science named lenacapavir its 2024 “Breakthrough of the Year.”

Yeztugo received FDA approval under Priority Review. Additionally, in October 2024, Yeztugo was granted Breakthrough Therapy Designation, which is intended to expedite the development and review of new drugs that may demonstrate substantial improvement over available therapy.

Gilead’s U.S. access strategy for Yeztugo is designed to enable broad uptake and availability for individuals with and without insurance coverage

In the U.S., Gilead is working closely with insurers, healthcare systems and other payers with the goal of ensuring broad insurance coverage for Yeztugo. Additionally, for eligible commercially insured individuals with commercial insurance, Gilead’s Advancing Access® Co-Pay Savings Program will reduce out-of-pocket costs to as little as zero dollars.

Gilead is also committed to helping to ensure that people without insurance in the U.S. will be able to benefit from Yeztugo. For those who are eligible, Gilead’s Advancing Access medication assistance program will provide Yeztugo free of charge.

https://en.wikipedia.org/wiki/Lenacapavir

FDA Approves Yeztugo (lenacapavir) as the First and Only HIV Prevention Option Offering 6 Months of Protection

FDA Approves Xifyrm (meloxicam) Injection for the Management of Moderate-to-Severe Pain in Adults

In continuation of update on Meloxicam

Azurity Pharmaceuticals, Inc.  announced the U.S. Food and Drug Administration (FDA)  approval of  Xifyrm (meloxicam injection) an IV non-steroidal anti-inflammatory drug (NSAID) that offers once daily dosing.

Xifyrm is indicated for use in adults for the management of moderate-to-severe pain, alone or in combination with non-NSAID analgesics. Xifyrm provides a non-opioid analgesic in a 30mg/mL vial for IV bolus injection over 15 seconds. Because of delayed onset of analgesia, Xifyrm alone is not recommended for use when rapid onset of analgesia is required.

“Xifyrm demonstrates our commitment to improving patient care by providing an alternate dosage form for pain management,” said Ron Scarboro, CEO at Azurity Pharmaceuticals. “Xifyrm addresses an important clinical need, especially for patients requiring a non-opioid component to multimodal analgesia strategies.”

Xifyrm will be available in the coming weeks. For full prescribing information, including boxed warning and safety profile, please visit www.Xifyrm.com

https://en.wikipedia.org/wiki/Meloxicam

FDA Approves Xifyrm (meloxicam) Injection for the Management of Moderate-to-Severe Pain in Adults