FDA Approves Arbli (losartan potassium) Oral Suspension as the First Ready-to-Use Oral Liquid Losartan in the U.S.

In continuation of my update on losartan

Scienture Holdings, Inc.   announced the U.S. Food and Drug Administration (FDA)  approval of SCN-102, one of the products being developed by Scienture, LLC, a wholly owned subsidiary of Scienture Holdings, Inc., with the brand name Arbli (losartan potassium) Oral Suspension, 10 mg/mL. Arbli is meant for the treatment of hypertension in patients greater than 6 years old, for the reduction of risk of stroke in patients with hypertension and left ventricular hypertrophy and for the treatment of diabetic nephropathy in certain patients with type 2 diabetes. Arbli is the first and only FDA approved ready-to-use oral liquid losartan in the U.S. market.

Arbli is a novel proprietary formulation of losartan, a proven therapy for treating hypertension, which provides a tailored approach to patients that require or prefer an oral liquid option of losartan. Appropriate dosing is now easier, safe and effective, while providing the assurance of quality as an FDA-approved product. Arbli provides a safe and convenient option to patients requiring a liquid formulation and addresses the intrinsic risks associated with potential inconsistencies in the process of crushing tablets to extemporaneously compound losartan prescriptions. Arbli has two issued patents from the USPTO, which are also expected to be listed in the FDA Orangebook.

Losartan is classified as an angiotensin receptor blocker (ARB) for treating hypertension and is one of the highest prescribed molecules for this indication. Current products in the market containing losartan are available only as oral solids, which can be further compounded to a liquid formulation. Arbli is the first liquid formulation of losartan on the market that does not require compounding and has reduced dosing volume and long-term shelf life at room temperature storage. IQVIA data (MAT December 2024) indicates a total annual sales of approximately $292 million and a prescription volume of 68 million (TRx) for losartan in the US market.

“The approval of Arbli exemplifies our deep commitment to develop high value products that address unique and underserved patient needs. A significant number of patients can benefit from a safe and efficacious ready-to-use oral liquid formulation of losartan. We are excited with the approval of our first brand product which is part of our upcoming pipeline of novel specialty products,” remarked Shankar Hariharan, CEO of Scienture, LLC.

“We are pleased to bring to market, Arbli, a transformative therapy option containing losartan, one of the most widely prescribed molecules in its class, to patients, caregivers and healthcare professionals (HCPs). We expect to commercially launch and make Arbli available to patients in the U.S. in Q3 2025,” said Narasimhan Mani, President of Scienture, LLC.

“This announcement is extremely exciting and is a clear demonstration of the value Scienture, LLC brings to the combined company and its shareholders,” said Suren Ajjarapu, Chairman of the Board, Scienture Holdings, Inc.

REF ;https://en.wikipedia.org/wiki/Losartan

FDA Approves HemiClor (chlorthalidone) for the Treatment of Hypertension

PRM Pharma, LLC  announced the U.S. Food and Drug Administration (FDA) approval of HemiClor (12.5 mg chlorthalidone) tablets for the treatment of hypertension in adults, to lower blood pressure.

Chlorthalidone, a thiazide-like diuretic, has been studied extensively over several decades in the treatment of hypertension. It is recognized in the 2017 ACC/AHA Guideline for the Prevention, Detection, Evaluation and Management of High Blood Pressure in Adults as the preferred diuretic based on its prolonged half-life and evidence of cardiovascular outcome benefits.1 Landmark studies such as the ALLHAT and SHEP trials, sponsored by the National Heart, Lung, and Blood Institute, demonstrated that stepped-care treatment strategies—which often began with 12.5 mg of chlorthalidone—were effective in reducing the risk of cardiovascular events.2,3 These findings support the use of 12.5 mg as the recommended starting dose in current hypertension treatment guidelines, either to initiate antihypertensive therapy or to serve as addon therapy when additional blood pressure reduction is needed.1,4

HemiClor now provides U.S. patients and healthcare professionals access to a 12.5 mg chlorthalidone tablet formulation that aligns with guideline recommendations. Until now, only higher-dose chlorthalidone tablets (25 mg and 50 mg) have been available in the U.S. This new low-dose option may help clinicians better individualize therapy and potentially reduce the risk of dose-related side effects. As noted in the approved prescribing information, metabolic adverse effects such as hypokalemia and hyperuricemia are dose-related; thus, a lower starting dose may help mitigate these risks when initiating treatment.5

“Chlorthalidone has played a key role in hypertension treatment strategies for decades,” said William B. White, M.D., Professor Emeritus at the University of Connecticut School of Medicine and past president of the American Society of Hypertension. “Having access to a 12.5 mg dose in the U.S. may offer clinicians additional flexibility when initiating therapy and aligns with current treatment recommendations for many adult patients with stage 1 or stage 2 hypertension.”

Joseph T. McDevitt, President and CEO of PRM Pharma, LLC, added, “We are proud to introduce HemiClor as a new treatment option for adults with hypertension. Our mission is to address unmet clinical needs by developing low-dose pharmaceutical products that support evidence-based care. Lower effective doses may offer a more individualized approach to initiating therapies, particularly for elderly patients who are more susceptible to dose-related adverse effects.”

HemiClor (12.5 mg chlorthalidone tablets) is expected to be available in pharmacies nationwide beginning in May 2025.

Indication and Usage

HemiClor (chlorthalidone) is indicated for the treatment of hypertension to lower blood pressure. Lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions. These benefits have been demonstrated in controlled trials of antihypertensive drugs from a wide variety of pharmacologic classes, including chlorthalidone.

REF: https://en.wikipedia.org/wiki/Chlortalidone

FDA Approves Blujepa (gepotidacin) for the Treatment of Uncomplicated Urinary Tract Infections

GSK plc  announced the US Food and Drug Administration (FDA) approval of Blujepa (gepotidacin) for the treatment of female adults (≥40 kg) and paediatric patients (≥12 years, ≥40 kg) with uncomplicated urinary tract infections (uUTIs) caused by the following susceptible microorganisms: Escherichia coli, Klebsiella pneumoniae, Citrobacter freundii complex, Staphylococcus saprophyticus and Enterococcus faecalis.

Discovered by GSK scientists, Blujepa is a first-in-class oral antibiotic with a novel mechanism of action that is part of GSK’s infectious diseases portfolio.

Tony Wood, Chief Scientific Officer, GSK, said: “The approval of Blujepa is a crucial milestone with uUTIs among the most common infections in women. We are proud to have developed Blujepa, the first in a new class of oral antibiotics for uUTIs in nearly three decades, and to bring another option to patients given recurrent infections and rising rates of resistance to existing treatments.”

uUTIs are the most common infection in women, impacting up to 16 million women in the US annually.1-4 Over half of all women are affected by uUTI in their lifetime,5 with approximately 30% suffering from at least one recurrent episode which can cause significant patient burden, including discomfort and restriction of daily activities.6 New treatments are needed as the number of uUTIs caused by drug-resistant bacteria is increasing which can result in higher treatment failure rates.7

Thomas Hooton, MD, Professor of Clinical Medicine, University of Miami School of Medicine said: “For many, uUTIs can be a burden that severely impacts daily life. With an increasing number of patients experiencing recurrent infections, there remains a clear need for continued research of antimicrobials to help address ongoing patient challenges and the strain on healthcare systems.”

The approval is based on positive results from the pivotal phase III EAGLE-2 and EAGLE-3 trials which demonstrated non-inferiority to nitrofurantoin, one of the leading current standard of care options for uUTI, in female adults (≥40 kg) and paediatric patients (≥12 years, ≥40 kg) with a confirmed uUTI. In EAGLE-2, Blujepa demonstrated non-inferiority in therapeutic success which occurred in 50.6% (162/320) of participants compared to 47.0% (135/287) for nitrofurantoin (covariate-adjusted treatment difference 4.3%, 95% CI (-3.6, 12.1)). In EAGLE-3, Blujepa demonstrated statistically significant superiority versus nitrofurantoin (one-sided p-value 0.0003). Therapeutic success occurred in 58.5% (162/277) of participants compared to 43.6% (115/264) for nitrofurantoin (covariate-adjusted treatment difference 14.6%, 95% CI (6.4, 22.8)).

The safety and tolerability profile of Blujepa in the EAGLE-2 and EAGLE-3 phase III trials was consistent with previous trials. The most commonly reported adverse events (AEs) in Blujepa participants were gastrointestinal (GI). Diarrhoea was the most common (16% of participants), followed by nausea (9%). Of the participants who reported GI AEs in the Blujepa group, the most common maximum severity was mild (69% Grade 1) and moderate (28% Grade 2). Participants with Grade 3 GI events accounted for 3% of all patients with GI events and occurred in <1% of all participants. There was one drug-related serious adverse event in each treatment arm (Blujepa and nitrofurantoin) across the two trials.

REF: https://en.wikipedia.org/wiki/Gepotidacin#:~:text=in%20March%202025.-,Names,under%20the%20brand%20name%20Blujepa.

FDA Approves Vykat XR (diazoxide choline) to Treat Hyperphagia in Prader-Willi Syndrome

Soleno Therapeutics, Inc.  announced  the U.S. Food and Drug Administration (FDA)  approval of  Vykat XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS). Soleno expects Vykat XR to be available in the U.S. beginning in April 2025.

“The approval of Vykat XR is a significant milestone for Soleno and, most importantly, for the PWS community who have had no options to treat the most disruptive aspect of this disease,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. “We are deeply grateful to the many individuals with PWS, their caregivers and clinical sites who participated in our trials, the advocacy groups, including FPWR and PWSA | USA, the advocates who have tirelessly supported the approval of Vykat XR, the FDA for a collaborative review process, and our employees who have been committed to delivering Vykat XR to those with PWS.”

“The FDA approval of Vykat XR is an incredible achievement for the entire PWS community,” said Jennifer Miller, M.D., Professor of Pediatric Endocrinology at the University of Florida, Gainesville, who specializes in treating children and adults with PWS and is a principal investigator in the Vykat XR clinical development program. “I am excited to have Vykat XR available to help treat hyperphagia, which is the most life-limiting aspect of PWS. Families of people with PWS have been prisoners in their own homes because of the need to provide constant, eyes-on supervision 24/7 with access to food being completely restricted.”

“Today marks a historic day for the PWS community. The FDA’s approval of Vykat XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families,” said Stacy Ward, Chief Executive Officer of the Prader-Willi Syndrome Association | USA. “Our families experience the constant and disruptive challenges of hyperphagia, and Vykat XR offers hope to so many.”

"This approval is a testament to the power of persistence, science, and advocacy," said Susan Hedstrom, Executive Director of the Foundation for Prader-Willi Research. "For years, families and researchers have worked towards a treatment option that truly addresses the complexities of PWS. Today, we take a major step forward in changing the future for individuals navigating hyperphagia associated with PWS."

The FDA approval of Vykat XR was based on an adequate and well-controlled study and safety data from the comprehensive clinical development program. Efficacy was established during the 16-week randomized withdrawal study period of Study 2-RWP (Study C602-RWP), a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial. Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with individuals who remained on Vykat XR. Prior to participating in the randomized withdrawal period, all individuals received double-blind and/or open-label Vykat XR for a mean duration of 3.3 years.

Vykat XR has a well-established safety profile with over four years of data across four double-blind and/or open label studies. The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to 10% of individuals receiving Vykat XR and at 2% greater than placebo included hypertrichosis, edema, hyperglycemia and rash.

Ref: https://en.wikipedia.org/wiki/Diazoxide#:~:text=Diazoxide%2C%20used%20as%20the%20salt,available%20as%20a%20generic%20medication.

FDA Grants Accelerated Approval for Vanrafia (atrasentan) for Proteinuria Reduction in Primary IgA Nephropathy

Novartis  announced the US Food and Drug Administration (FDA)  accelerated approval for Vanrafia® (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g1. Vanrafia is a once-daily, non-steroidal, oral treatment that can be added onto supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.

Vanrafia was granted accelerated approval based on a prespecified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo1. It has not been established whether Vanrafia slows kidney function decline in patients with IgAN. The continued approval of Vanrafia may be contingent upon the verification of clinical benefit from the ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 1361. The eGFR data are expected in 2026 and intended to support traditional FDA approval.

“Today’s approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,” said Richard Lafayette, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member. “Vanrafia is a selective ETA receptor antagonist that effectively reduces proteinuria, a major risk factor in IgAN. Taking early, decisive action is critical to help improve outcomes for these patients who too often progress toward kidney failure.”

IgAN is a progressive, rare kidney disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria10. With almost 13 out of every million people in the US diagnosed per year, it is one of the most common autoimmune kidney diseases, and each person’s journey is unique11,12. Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring maintenance dialysis and/or kidney transplantation3-10, and response to treatment can vary12,13. Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patients.

REF: https://en.wikipedia.org/wiki/Atrasentan#:~:text=in%20April%202025.-,Names,under%20the%20brand%20name%20Vanrafia.

FDA Approves Mezofy (aripiprazole) Oral Film for the Treatment of Schizophrenia

In continuation of my update on Aripiprazole

CMG Pharmaceuticals, an affiliate of Cha Biotech, announced on the 16th that it had received product approval for the schizophrenia treatment drug Mezofy (formerly Depibzo) from the U.S. Food and Drug Administration (FDA).

Mezofy is an oral film-type schizophrenia treatment drug (ingredient name: aripiprazole) developed by CMG Pharmaceuticals. Patients with mental illnesses including schizophrenia often refuse or spit out medication, but the film-type drug can be taken without water and easily dissolves in the mouth, solving this problem.

CMG Pharmaceuticals first applied for FDA approval in December 2019. At that time, CMG Pharmaceuticals was the world’s first film-type schizophrenia treatment company to knock on the FDA’s door. However, the supplementary inspection was delayed due to problems with overseas raw material factories and the impact of the COVID-19 pandemic.

Meanwhile, Opipza, aripiprazole film treatment from China's LP Pharma, received FDA approval in July of last year. CMG Pharmaceuticals then reapplied for product approval in October of last year and received marketing approval.

Mezofy is the fourth product from a domestic pharmaceutical company to receive FDA approval for an improved new drug. An improved new drug is not simply a copy of a new drug whose patent has expired, but rather an improvement in the dosage method by changing the efficacy or formulation. Unlike simple generic drugs, it is protected separately by a patent.

The company explained, “The price of improved new drugs is set higher than that of generic drugs, and they can be marketed and prescribed under the product name rather than the ingredient name, which is advantageous in increasing awareness in the market.”

CMG Pharmaceuticals expects Mezofy to be launched in the US in the first half of 2026. According to Data Monitor, a US market research and analysis firm, the US schizophrenia treatment market is the largest in the world, at 12 trillion won.

The company predicted that the market for Mezofy would expand to over KRW 22 trillion when its indications are expanded to include bipolar disorder, major depressive disorder, autism spectrum disorder, and Tourette's disorder.

The company said, “We plan to complete the selection of a local distribution partner in the U.S. by the second half of this year,” and “We will work with our distribution partners to establish a competitive drug pricing strategy.”

CMG Pharmaceuticals has set a goal of achieving annual sales of over 100 billion won within five years of entering the U.S. market. CMG Pharmaceuticals CEO Lee Ju-hyung said, “After proving the excellence of Mezofy in the U.S. market, we will advance into other overseas markets.”

At the time of the initial application for product approval, the product name was Depipzo. The company explained that in collaboration with the global pharmaceutical branding specialist Brand Institute, it decided on Mezofy as a name that is easier to remember and less likely to cause prescription errors.

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

FDA Approves Atzumi (dihydroergotamine) Nasal Powder for the Acute Treatment of Migraine

Shin Nippon Biomedical Laboratories, Ltd. (TSE:2395),  announced  the U.S. Food and Drug Administration (FDA) approval of    New Drug Application (NDA) for Atzumi™(dihydroergotamine (DHE)) nasal powder for the acute treatment of migraine with or without aura in adults. Atzumi was previously known as STS101.

 

Migraine is a neurological disorder that is thought to be the result of temporary changes in the chemicals, nerves and blood vessels in the brain, with symptoms that are often incapacitating. According to the American Migraine Foundation, approximately 40 million Americans live with migraine. It is the second leading cause of disability worldwide in terms of time lost to disability and most common cause of disability among young women.

"The approval of Atzumi is a milestone to celebrate, providing a new option for the acute treatment of migraine combining long-proven benefits of DHE with a patient-friendly and easy-to-use delivery system developed based on SNBL's novel intranasal drug delivery platform technology," said Dr. Ryoichi Nagata, President and CEO of Satsuma. "We believe that Atzumi will contribute to improving the quality of life of patients struggling for relief from these highly disabling problems."

"DHE plays a unique clinical role in the acute treatment of migraine, providing patients long lasting effects and the unique ability to provide benefit even when taken late in a migraine attack. The convenience of Atzumi, the only DHE nasal powder, will offer patients ease of use combined with the important known DHE clinical advantages", said Dr. Stewart J. Tepper, M.D., Vice President of the New England Institute for Neurology and Headache in Stamford, Connecticut.

About Atzumi

Atzumi is a proprietary drug device product incorporating both Satsuma's advanced nasal powder formulation of dihydroergotamine (DHE) administered via its unique nasal delivery device. The product is designed to provide patients an easy-to-use and easy-to-carry treatment option.

The FDA approval for Atzumi is based on two clinical studies (Phase 1 PK trial and ASCEND Phase 3 open-label, long-term safety trial), which demonstrated fast absorption, rapid achievement of high DHE plasma concentrations, and sustained DHE plasma levels over time as well as safety and tolerability in subjects with migraine.

About Dihydroergotamine (DHE)

Since its approval in 1946, DHE has long been recommended in published migraine treatment guidelines as a first-line acute treatment option for migraine and has significant advantages versus other anti-migraine treatments for many patients. However, disadvantages of current DHE liquid nasal spray and injectable products, including invasive and burdensome administration and/or sub-optimal clinical performance, have limited the widespread use of DHE.

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

https://pubchem.ncbi.nlm.nih.gov/compound/Dihydroergotamine#section=2D-Structure

FDA Approves Tepylute (thiotepa) Ready-to-Dilute Injectable Formulation to Treat Breast Cancer and Ovarian Cancer

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for Tepylute, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for Tepylute, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.1

‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.”

Tepylute, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market.

“The approval of Tepylute represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.

Tepylute is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2

“Among Tepylute’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for Tepylute in the near future.”

The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4

Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024.

‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.”

Tepylute, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market.

“The approval of Tepylute represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.

Tepylute is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2

“Among Tepylute’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for Tepylute in the near future.”

The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4

Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024.

FDA Approves Tepylute (thiotepa) Ready-to-Dilute Injectable Formulation to Treat Breast Cancer and Ovarian Cancer

FDA Approves Ohtuvayre (ensifentrine) for the Maintenance Treatment of Chronic Obstructive Pulmonary Disease (COPD)

Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), announces the US Food and Drug Administration (“FDA”) approved Ohtuvayre (ensifentrine) for the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adult patients. Ohtuvayre is the first inhaled product with a novel mechanism of action available for the maintenance treatment of COPD in more than 20 years.

Ohtuvayre is a first-in-class selective dual inhibitor of the enzymes phosphodiesterase 3 and phosphodiesterase 4 (“PDE3 and PDE4”) that combines bronchodilator and non-steroidal anti-inflammatory effects in one molecule. Ohtuvayre is delivered directly to the lungs through a standard jet nebulizer without the need for high inspiratory flow rates or complex hand-breath coordination.

“The approval of Ohtuvayre is a significant advance in COPD care, and we believe Ohtuvayre’s novel profile can change the treatment paradigm for COPD,” said David Zaccardelli, Pharm. D., President and Chief Executive Officer of Verona Pharma. “We plan to launch Ohtuvayre in the third quarter 2024, ensuring Ohtuvayre is available to help the millions of patients who still experience daily COPD symptoms.”

Michael Wells, MD, Associate Professor in the Division of Pulmonary, Allergy, and Critical Care Medicine at the University of Alabama Birmingham, commented: “In my experience, despite maintenance therapy, most patients report grappling with daily symptoms, including breathlessness and persistent coughing. COPD has a significant impact on both mortality and morbidity in the US, and until today, innovation in inhaled treatment modalities has been limited to combinations of existing treatment classes for over two decades. Ohtuvayre, as a first-in-class PDE3 and PDE4 inhibitor, offers a needed, unique approach and is an important advance in the treatment of COPD.”

The US approval of Ohtuvayre was based on extensive data including the Phase 3 ENHANCE trials, the results of which were published in the American Journal of Respiratory and Critical Care Medicine. In the ENHANCE trials, Ohtuvayre demonstrated clinical benefits both alone and when used with other maintenance therapies. Ohtuvayre was well-tolerated in a broad population of subjects with moderate to severe COPD.

The Company is fully staffed to launch and expects Ohtuvayre to be available in the third quarter 2024 through an exclusive network of accredited specialty pharmacies.

About Ohtuvayre (ensifentrine)

Ohtuvayre is the first inhaled therapy for the maintenance treatment of COPD that combines bronchodilator and non-steroidal anti-inflammatory activities in one molecule. Verona has evaluated nebulized Ohtuvayre in its Phase 3 clinical program ENHANCE (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) for COPD maintenance treatment. Ohtuvayre met the primary endpoint in both ENHANCE-1 and ENHANCE-2, demonstrating statistically significant and clinically meaningful improvements in lung function. A fixed-dose combination of ensifentrine and glycopyrrolate, a LAMA, is currently under development for the maintenance treatment of COPD. Ensifentrine has potential applications for development in non-cystic fibrosis bronchiectasis, cystic fibrosis, asthma and other respiratory diseases.

REF: https://en.wikipedia.org/wiki/Ensifentrine

FDA Approves Ohtuvayre (ensifentrine) for the Maintenance Treatment of Chronic Obstructive Pulmonary Disease (COPD)

FDA Approves Sofdra (sofpironium) Topical Gel for the Treatment of Primary Axillary Hyperhidrosis

Clinical dermatology company, Botanix Pharmaceuticals Ltd. (ASX:BOT, Botanix or the Company), is pleased to announce the US Food and Drug Administration (FDA) approval of Sofdra™ (sofpironium) gel, 12.45%. Sofdra is a prescription medicine used to treat primary axillary hyperhidrosis (excessive underarm sweating) in adults and children 9 years and older.

Sofdra is the first and only new chemical entity approved by the FDA to treat primary axillary hyperhidrosis and presents a novel safe and effective solution for patients who have lacked treatment options for this socially challenging medical condition.

Botanix Chief Executive Officer, Dr Howie McKibbon, commented: "We are pleased to share this accomplishment with our dedicated Botanix team and dermatologist partners, patients who participated in the clinical studies and our shareholders who made this approval possible."

"This is a transformative event for Botanix as we transition from a development stage to a revenue generating dermatology company."

Hyperhidrosis is a condition characterised by abnormally increased sweating, beyond that required to regulate body temperature.1 The disproportionate sweat production that characterizes hyperhidrosis, results in a disabling medical condition with profound effects on the patient’s quality of life. Hyperhidrosis affects work productivity, daily routine activities, emotional well-being and personal relationships.2 Hyperhidrosis is the third largest dermatology condition (after acne and atopic dermatitis), with approximately 10 million patients in the US with primary axillary hyperhidrosis.3

David Pariser, MD, leading expert on hyperhidrosis, founding board member of the International Hyperhidrosis Society and past President of the American Academy of Dermatology commented: "The approval of Sofdra is terrific news for the hyperhidrosis community, which has been frustrated by the lack of effective and convenient treatment options."

"The availability of a new treatment alternative that is topical, well-tolerated, effective and easy to use is truly exciting and would be welcomed amongst patients and physicians."

The FDA approval of Sofdra was supported by results from the two pivotal Phase 3 ‘CARDIGAN’ studies which evaluated the efficacy and safety of Sofdra versus vehicle and enrolled 701 patients with primary axillary hyperhidrosis. In the studies, treatment with Sofdra successfully met all primary and secondary endpoints with clinically and statistically meaningful changes from baseline in Gravimetric Sweat Production (GSP) and the Hyperhidrosis Disease Severity Measure-Axillary, 7-item (HDSM-AX7) score.

An early patient experience program is planned to be launched by the Company in Q3 CY2024 to enable highly qualified patients to gain early access to Sofdra. These patients will be guided through the telemedicine and payer reimbursement process to be the first commercial users of the product. Broader launch of Sofdra is expected to follow in early Q4 CY2024 and Botanix expects to receive first revenues from sales in Q4 CY2024.

Botanix Executive Chairman, Mr Vince Ippolito, commented: "We are very excited to provide a new option for the 10 million patients with primary axillary hyperhidrosis in the United States."

"As the first and only new chemical entity, Sofdra represents a new therapeutic approach for dermatologists to treat patients with this disabling medical condition."

REF: https://en.wikipedia.org/wiki/Sofpironium_bromide

FDA Approves Sofdra (sofpironium) Topical Gel for the Treatment of Primary Axillary Hyperhidrosis

FDA Approves Vigafyde (vigabatrin) as the First and Only Ready-to-Use Vigabatrin Oral Solution

Pyros Pharmaceuticals, Inc. (Pyros or the Company), a leader in the development of enhanced specialty pharmaceuticals for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved Vigafyde™, the only ready-to-use vigabatrin oral solution. Vigafyde™ (vigabatrin) oral solution, is indicated as a monotherapy for the treatment of pediatric patients 1 month to 2 years of age with infantile spasms (IS), where the potential benefits outweigh the potential risk of vision loss.  

Infantile spasms, a rare but severe form of epilepsy, poses significant challenges for patients and their families. Pyros is committed to providing dependable services and an affordable treatment option to help ensure that families facing an IS diagnosis, and whose children are prescribed vigabatrin can quickly obtain this essential therapy.

"The approval of Vigafyde™ marks the first new drug indicated for infantile spasms in fifteen years and underscores our unwavering commitment to supporting families facing this challenging condition," stated Michael Smith, co-founder and Chief Executive Officer of Pyros.

“This is a momentous day for patients and caregivers who have long awaited the first ready-to-use vigabatrin. I believe this approval will bring renewed energy and momentum to the infantile spasms community, enhancing our efforts to improve disease education, develop care pathways, and expand investment in infantile spasms research,” stated Edwin Urrutia, co-founder and Chief Operating Officer at Pyros.

Vigafyde™ is expected to be available in the second half of 2024. Additionally, Pyros Total Care™, the Company’s personalized comprehensive support program, is available to assist families throughout the treatment journey.

About Pyros Total Care™

As part of the Company’s commitment to prioritizing patient access to treatments for those who need them most, Pyros provides ongoing personalized support to caregivers through Pyros Total Care™ for those prescribed Vigafyde™. The support program offers personal assistance and financial resources to caregivers whose child is starting or continuing therapy.

Our dedicated support team includes a nurse educator, reimbursement support, and a clinical pharmacist. For more information, visit PyrosTotalCare.com or call 1-888-760-8330, Monday to Friday, 8 a.m. to 5 p.m. Central Time.

About Infantile Spasms

Infantile spasms (IS) is a rare, severe form of epilepsy that typically begins in children less than one year old.1 Infantile spasms can appear as subtle but repetitive movements that can often be overlooked or misdiagnosed. IS can lead to long-term permanent issues such as continued seizures, other forms of epilepsy, autism spectrum disorder, and developmental issues.2 The American Academy of Neurology conducted a systematic review of treatment for IS and concluded that successful treatment of IS can improve the long-term prognosis.3

About Vigabatrin

Vigabatrin is a medication used in the treatment of infantile spasms and is designed to inhibit the enzyme GABA transaminase, consequently increasing gamma-aminobutyric acid (GABA) levels in the brain.4,5 This release is thought to enhance seizure control for patients by modulating neuronal excitability.6 Vigabatrin's mechanism of action underscores its importance in treating seizure disorders.7 Its impact may extend beyond seizure management, with emerging research indicating potential benefits in mitigating neurodevelopmental complications associated with certain epilepsy syndromes

FDA Approves Vigafyde (vigabatrin) as the First and Only Ready-to-Use Vigabatrin Oral Solution

FDA Grants Accelerated Approval to Iqirvo (elafibranor) for the Treatment of Primary Biliary Cholangitis

Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo (elafibranor) 80 mg tablets for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. Iqirvo may be prescribed immediately in the U.S. for eligible patients.

This indication is approved under accelerated approval based on the reduction of alkaline phosphatase (ALP). Improvement in survival or prevention of liver decompensation events has not been demonstrated. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Iqirvo is not recommended for people who have or develop decompensated cirrhosis (e.g., ascites, variceal bleeding, hepatic encephalopathy).

“For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC. Left unmanaged, PBC can progress, leading to liver failure and in some cases, the need for a liver transplant,” said Christelle Huguet, Executive Vice President, Head of Research and Development at Ipsen. “Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone. Iqirvo is, therefore a much-needed treatment option and the first new medicine for PBC in nearly a decade.”

Iqirvo is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. Iqirvo was in-licensed from GENFIT in 2021. The accelerated approval of Iqirvo is based on data from the Phase III ELATIVE trial published in the New England Journal of Medicine1. The ELATIVE trial demonstrated that 13 times more patients achieved the composite primary endpoint of biochemical response when treated with Iqirvo plus UDCA (n=108) versus placebo plus UDCA (=53) (respectively 51% versus 4% for a 47% treatment difference). ALP is a biochemical marker and is used as a surrogate endpoint in PBC trials. Secondary endpoints showed normalization in ALP levels in only Iqirvo-treated patients (15% for Iqirvo plus UDCA (n=108) versus 0% for placebo plus UDCA (n=53)). Most patients (95%) received study treatment (Iqirvo or placebo) in combination with UDCA.

The most common adverse reactions with Iqirvo reported in ≥10% of study participants were weight gain, abdominal pain, diarrhea, nausea, and vomiting. Some study participants treated with Iqirvo experienced myalgia, myopathy and rhabdomyolysis; fractures; adverse effects on fetal and newborn development; drug-induced liver injury; hypersensitivity reactions; or biliary obstruction. See full Important Safety Information below.

“Data from the pivotal Phase III ELATIVE clinical trial demonstrated that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit and risk data,” said Dr. Kris Kowdley, Director at Liver Institute Northwest, Washington, and a primary investigator on the ELATIVE study. “The approval of Iqirvo will allow healthcare providers in the U.S. to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”

PBC is a rare, autoimmune, cholestatic liver disease where a build-up of bile and toxins (cholestasis) and chronic inflammation causes irreversible fibrosis (scarring) of the liver and destruction of the bile ducts. Impacting approximately 100,000 people in the U.S.2, the majority being women, PBC is a lifelong condition that can worsen over time if not effectively treated, leading to liver transplant and in some cases, premature death. PBC also affects day-to-day life, with people most commonly experiencing severe fatigue symptoms and debilitating itch (pruritus).

“People living with PBC can feel like the symptoms they experience are dismissed by family members, friends, or even their doctors because they have not experienced something similarly disruptive in their lives. People with PBC may also feel uncertainty around the disease progression and if, or when, their liver health may deteriorate,” said Carol Roberts, Executive President of PBCers, a patient advocacy organization in the U.S. providing support to people living with PBC. “Earlier diagnosis and education about PBC, along with new treatment options are important to meet the current needs of people living with PBC.”

Iqirvo has been submitted to the European Medicines Agency (EMA) and the UK Medicines and Healthcare Products Regulatory Agency (MHRA), seeking authorization for PBC, with final EMA and MHRA regulatory decisions anticipated in the second half of 2024. The FDA approval of Iqirvo further strengthens Ipsen’s portfolio of treatments for rare cholestatic liver diseases available to patients in the U.S. This includes our FDA-approved medicine for the treatment of pruritus in patients three months and older with progressive familial intrahepatic cholestasis (PFIC) and for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome (ALGS).

REF : https://en.wikipedia.org/wiki/Elafibranor

FDA Grants Accelerated Approval to Iqirvo (elafibranor) for the Treatment of Primary Biliary Cholangitis

FDA Approves Lumisight (pegulicianine) Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy

Lumicell, Inc., a privately held company focused on developing innovative fluorescence-guided imaging technologies for cancerous tissue detection during surgery, today announced the U.S. Food & Drug Administration (FDA) approved the company’s New Drug Application (NDA) for its Lumisight™ (pegulicianine) optical imaging agent and its Premarket Approval (PMA) application for Lumicell™ Direct Visualization System (DVS), together referred to as LumiSystem™.

With 84% diagnostic accuracy, LumiSystem enables surgeons to scan the breast cavity post-lumpectomy, in real-time, to detect and resect residual cancer that may have otherwise been missed, potentially sparing some patients from second surgeries.1-2 The LumiSystem combination is indicated for fluorescence imaging in adults with breast cancer as an adjunct for the intraoperative detection of cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery.

“We are immensely proud of the dual approval of Lumisight and Lumicell DVS – we believe this is the first drug-device combination product approved in over a decade to have followed both of the FDA's most stringent NDA and PMA review processes,”3 said Howard Hechler, President and Chief Operating Officer, Lumicell. “With the FDA’s approval, LumiSystem is now the first and only imaging combination product capable of detecting cancerous tissue where it matters most, inside the breast cavity.”

“Breast cancer is all too common, and sadly, 1 in 8 women will develop it during their lifetime,”4 said Kelly Hunt, MD, Chair of the Department of Breast Surgical Oncology at MD Anderson Cancer Center and President of the Society of Surgical Oncology. “Our most common surgical procedure to treat these women is lumpectomy. Unfortunately, the intraoperative tools we have are limited and do not identify the extent of tumor accurately enough, making it challenging to achieve a complete tumor resection, leading to as many as 36% of patients needing a second surgery.”5

“Up to 65% of the time, we do not find residual cancer in the second surgery and are left wondering if we performed an unnecessary surgery due to a false positive margin assessment or if the cancer was missed again in the second surgery,”6 said Irene Wapnir, MD, Breast Surgical Oncologist and Professor of Surgery, Stanford University School of Medicine.

“During lumpectomy surgery, surgeons still struggle to identify and remove all of the tumor during the first operation,”7 said Barbara Smith, MD, PhD, Director of the Breast Program at Massachusetts General Hospital and Professor of Surgery at Harvard Medical School. “With LumiSystem, we will now have a technology that is clinically proven to achieve a more complete cancer resection during lumpectomy that could help some patients avoid a second surgery.”2

The safety of the system was established using data from more than 700 breast cancer patients across five clinical studies at top academic and regional community cancer centers across the U.S. The most common side effects with Lumisight are hypersensitivity and an abnormal color in urine. Lumisight may cause serious hypersensitivity reactions, including anaphylaxis. Results of the Investigation of Novel Surgical Imaging for Tumor Excision (INSITE) pivotal trial, used to support the efficacy of the system, were published in NEJM Evidence.

“We want to thank our clinical investigators and the hundreds of women who participated in our breast program for Lumisight and Lumicell DVS,” said Jorge Ferrer, Chief Scientific Officer, Lumicell. “Due to your important contributions, Lumisight and Lumicell DVS are now approved and will be available in the United States shortly.”

FDA Approves Lumisight (pegulicianine) Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy

FDA Approves Xromi (hydroxyurea) Oral Solution for Use in Pediatric Patients with Sickle Cell Anemia

The U.S. Food and Drug Administration (FDA) has approved Xromi, an oral solution formulation of hydroxyurea indicated to reduce the frequency of painful crises and reduce the need for blood transfusions in pediatric patients aged 6 months of age to less than 2 years with sickle cell anemia with recurrent moderate to severe painful crises.

Sickle cell anemia is caused by an abnormal version of hemoglobin called hemoglobin S, which leads to sickle-shaped red blood cells that can form painful clumps inside the blood vessels. These painful episodes are called sickle cell crises, and are one of the most common and distressing symptoms of sickle cell disease.

Hydroxyurea has been shown to reduce the frequency of painful episodes associated with sickle cell disease. It is thought to work by increasing levels of hemoglobin F (also called fetal hemoglobin because it is present in newborn babies) to make the red blood cells bigger, rounder, more flexible, and less likely to turn into a sickle shape.

Hydroxyurea was first approved in 1967 in an oral capsule formulation as a cancer treatment and has been used by doctors to treat sickle cell anemia since the 1980s. It was approved by the FDA for treating adults with sickle cell anemia in 1998, and children in 2017.

Xromi is supplied as a strawberry-flavored oral solution containing 100 mg/mL hydroxyurea. The Xromi package contains two dosing oral dosing syringes (a red oral dosing syringe graduated to 3 mL and a white oral dosing syringe graduated to 12 mL) for accurate measurement of the prescribed dose.

FDA Approves Xromi (hydroxyurea) Oral Solution for Use in Pediatric Patients with Sickle Cell Anemia

FDA Approves Rytelo (imetelstat) for the Treatment of Adult Patients with Lower-Risk MDS with Transfusion-Dependent Anemia

Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced that the U.S. Food and Drug Administration (FDA) has approved Rytelo™ (imetelstat) for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

“With the approval and availability of Rytelo, we believe eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “The approval of Rytelo as the first telomerase inhibitor is a testament to the power of our science and the passion of our people to innovate in the field of blood cancer. As we celebrate today’s momentous milestone, I would like to thank the patients and families, advocates, clinicians, study coordinators and site personnel, scientists, and Geron employees and collaborators past and present whose participation was integral to this achievement and to supporting our transformation into a commercial company.”

Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue1. These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival2,3. There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.

Approval Based on Results from IMerge Phase 3 Clinical Trial

“For patients with lower-risk MDS and anemia who are transfusion dependent, we have very few options today and often cycle through available therapies, making the approval of Rytelo potentially practice changing for us,” said Rami Komrokji, MD, Vice Chair, Malignant Hematology Department, Moffitt Cancer Center, who was an investigator of the pivotal IMerge clinical trial. “What is exciting about Rytelo is the totality of the clinical benefit across LR-MDS patients irrespective of ring sideroblast status or high transfusion burden, including sustained and durable transfusion independence and increases in hemoglobin levels, all within a well-characterized safety profile of generally manageable cytopenias. The treatment goal for patients with LR-MDS and anemia is transfusion-independence and before today, this wasn’t possible for many patients.”

The FDA approval of Rytelo is based on results from the IMerge Phase 3 clinical trial, published in The Lancet 4. The IMerge trial met its primary and key secondary endpoints, with Rytelo demonstrating significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for at least eight consecutive weeks (Rytelo 39.8% [95% CI 30.9–49.3]; placebo 15.0% [7.1–26.6]; p<0.001) and for at least 24 weeks (Rytelo 28.0% [95% CI 20.1-37.0]; placebo 3.3% [95% CI 0.4-11.5]; p<0.001). RBC-TI was durable and sustained in the Rytelo treated population, with a median RBC-TI duration for 8-week responders and 24-week responders of approximately 1 year and 1.5 years, respectively.

In an exploratory analysis of Rytelo-treated patients achieving ≥8-week RBC-TI, median increases in hemoglobin were 3.6 g/dL for Rytelo and 0.8 g/dL for placebo. Clinically meaningful efficacy results were observed across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category.

In the IMerge trial, the safety profile of Rytelo was well-characterized with generally manageable and short-lived thrombocytopenia and neutropenia, which are familiar side effects for hematologists who are experienced with managing cytopenias. The most common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks. Cytopenias were generally manageable with dose modifications. The intravenous administration of Rytelo every four weeks aligns to routine blood count monitoring for these patients.

The most common adverse reactions (incidence ≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets (thrombocytopenia), decreased white blood cells, decreased neutrophils (neutropenia), increased aspartate aminotransferase (AST), increased alkaline phosphatase (ALP), increased alanine aminotransferase (ALT), fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache. Clinically relevant adverse reactions in < 5% of patients who received Rytelo included febrile neutropenia, sepsis, gastrointestinal hemorrhage, and hypertension.

REF: https://en.wikipedia.org/wiki/Imetelstat

FDA Approves Rytelo (imetelstat) for the Treatment of Adult Patients with Lower-Risk MDS with Transfusion-Dependent Anemia