Lykos Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for MDMA-Assisted Therapy for PTSD

Lykos Therapeutics (formerly MAPS Public Benefit Corporation) ("Lykos"), a company dedicated to transforming mental healthcare, announced that the U.S. Food and Drug Administration ("FDA") has accepted its new drug application ("NDA") for midomafetamine capsules ("MDMA") used in combination with psychological intervention, which includes psychotherapy (talk therapy) and other supportive services provided by a qualified healthcare provider for individuals with post-traumatic stress disorder ("PTSD"). The FDA has granted the application priority review and has assigned a Prescription Drug User Fee Act ("PDUFA") target action date of August 11, 2024. If approved, this would be the first MDMA-assisted therapy and psychedelic-assisted therapy.

"Securing priority review for our investigational MDMA-assisted therapy is a significant accomplishment and underscores the urgent unmet need for new innovation in the treatment of PTSD," said Amy Emerson, chief executive officer of Lykos Therapeutics. "We remain focused on working with the FDA through the review process and preparing for a controlled launch with an emphasis on quality should this potential treatment be approved."

The NDA submission included results from numerous studies including two randomized, double-blind, placebo-controlled Phase 3 studies (MAPP1 and MAPP2) evaluating the efficacy and safety of MDMA used in combination with psychological intervention versus placebo with therapy in participants diagnosed with severe or moderate to severe PTSD, respectively. Both MAPP1 and MAPP2 studies met their primary and secondary endpoints and were published in Nature Medicine. 1, 2 The primary endpoint for both studies was to assess changes in PTSD symptom severity as measured by the change from baseline in Clinician-Administered PTSD Scale for DSM-5 ("CAPS-5"). The key secondary endpoint of both studies was to assess improvement in functional impairment associated with PTSD as measured by the change from baseline in the Sheehan Disability Scale ("SDS"). No serious adverse events were reported in the MDMA group in either study.

The FDA grants priority review for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. 

MDMA-assisted therapy has not been approved by any regulatory agency. The safety and efficacy of MDMA-assisted therapy has not been established for the treatment of PTSD. Investigational MDMA-assisted therapy is also being studied in other indications.

About MDMA-Assisted Therapy

MDMA (3,4-methylenedioxymethamphetamine) is not new to mental health professionals. MDMA is an entactogen— a class of psychoactive compounds that are differentiated from classic psychedelics (i.e., psilocybin, mescaline and LSD) and are defined based on their mechanism and known effects of increasing self-awareness leading to introspection and personal reflection.27,,28 In the 1970's and early 1980's MDMA was used in conjunction with talk therapy by mental health providers to enhance patients' access, processing, and communication of difficult emotions and experiences.29 In 1985, the U.S. Drug Enforcement Administration ("DEA") made MDMA a Schedule I drug under the Controlled Substances Act preventing it from being used for recreational or medical use. 30 Since then, research has shown the unique properties of MDMA allow it to act as a powerful catalyst to support psychotherapy by helping diminish the brain's fear response allowing people to access and process painful memories without being overwhelmed. 31 However, additional clinical trials would be needed to secure regulatory review and potential approval.

Lykos, with longstanding roots in advocacy for psychedelic medicine, pioneered the first randomized, double-blind, placebo controlled clinical trials evaluating the efficacy and safety of MDMA-assisted therapy as an investigational modality using midomafetamine (MDMA) in combination with psychological intervention to treat PTSD.

With a growing body of evidence supporting the potential medical use of MDMA, in 2017 the FDA granted the company's investigational MDMA-assisted therapy Breakthrough Therapy designation, a process designed to expedite the development and review of drugs intended to treat serious conditions for which preliminary scientific evidence indicates that it may demonstrate a substantial improvement over available therapies. If approved by the FDA, the U.S. Drug Enforcement Administration ("DEA") would be required to reschedule MDMA making it available for prescription medical use.

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

Lykos Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for MDMA-Assisted Therapy for PTSD

Blood Test to Predict Schizophrenia Shows Promise

Researchers say they have developed a blood test for schizophrenia.

More than 3 million people in the United States have schizophrenia, a disorder marked by hallucinations and delusions, or a related psychotic illness.

The new test, which is expected to be available later this year from MindX Sciences, identifies markers in the blood that objectively measure a person's risk for schizophrenia, allowing doctors to tailor treatments to their individual biology.

"Schizophrenia is hard to diagnose, especially early on, and matching people to the right treatment from the beginning is very important," said senior study author Dr. Alexander Niculescu, a professor of psychiatry and medical neuroscience at Indiana University School of Medicine in Indianapolis.

"Psychosis usually manifests in young adulthood -- a prime period of life," he explained in a university news release. "Stress and drugs, including marijuana, are precipitating factors on a background of genetic vulnerability. If left unchecked, psychosis leads to accumulating biological damage, social damage and psychological damage."

His team published its research Feb. 8 in the journal Molecular Psychiatry.

For their study, they followed psychiatric patients for more than a decade, identifying biomarkers that predicted high rates of hallucination and delusions, as well as future related hospitalizations. They also examined which biomarkers were targets of existing drugs.

The work builds on previous studies by Niculescu, who is also a staff psychiatrist at the Veterans Administration Medical Center in Indianapolis, and his colleagues over the past two decades.

They have examined blood biomarkers for other psychiatric issues, including anxiety, post-traumatic stress disorder (PTSD), memory disorders and suicide risk.

In general, Niculescu said, the best biomarkers were better predictors than standard scales used to evaluate someone who has hallucinations or delusions.

"Fortunately, biologically some of the existing medications work quite well if initiated early in the right patients," he said. "Social support is also paramount, and once that and medications are in place, psychological support and therapy can help as well."

While Niculescu said there is still much to learn, there is reason for optimism in this era of emerging precision psychiatry.

https://medicine.iu.edu/news/2024/02/psychosis-blood-test

Blood Test to Predict Schizophrenia Shows Promise 

Liquidia Corporation Provides Update on New Drug Application for Yutrepia (treprostinil) inhalation powder

Liquidia Corporation (the Company) (NASDAQ: LQDA) announced  the U.S. Food and Drug Administration (FDA) providing  an update on its review of the New Drug Application (NDA) for Yutrepia™ (treprostinil) inhalation powder to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). The FDA informed the Company that it is confirming the process for adding the PH-ILD indication as an amendment to the NDA for Yutrepia. Accordingly, the FDA is not able to issue an action letter in time to meet the previously issued Prescription Drug User Fee Act (PDUFA) goal date of January 24, 2024, and their review remains ongoing. The FDA did not request any additional clinical data to support the NDA and did not issue a new PDUFA goal date.

Dr. Roger Jeffs, Chief Executive Officer, said: “We are in active communication with the FDA regarding the process we followed to amend our NDA to add PH-ILD to the labeled indication. Whether the NDA is amended or supplemented, we will continue to prepare for the final FDA approval of Yutrepia to treat both PAH and PH-ILD patients following the expiration of regulatory exclusivity for Tyvaso® on March 31, 2024. As communicated by the tentative approval to treat PAH, Yutrepia has already met the regulatory standards for quality, safety and efficacy. We remain committed to addressing the unmet needs across all patients whose lives may be improved by the unique benefits of Yutrepia.”

On November 5, 2021, the FDA issued a tentative approval for Yutrepia for the treatment of PAH to improve exercise ability in adult patients with New York Heart Association (NYHA) Functional Class II-III symptoms. In July 2023, Liquidia filed an amendment to its New Drug Application for Yutrepia, seeking to add PH-ILD to the label. The FDA previously confirmed that Yutrepia may include the treatment of PH-ILD to the proposed label for Yutrepia without additional clinical studies.

Yutrepia also remains subject to ongoing litigation. Liquidia filed a request for Judge Andrews of the U.S. District Court for the District of Delaware (District Court) to set aside the injunction that was instituted in August 2022 tied to litigation filed by United Therapeutics (UTHR) alleging patent infringement of U.S. Patent No. 10,716,793 (the ‘793 Patent) in Case No. 1:20-cv-00755-RGA (the Original Hatch-Waxman Litigation). On December 20, 2023, the United States Court of Appeals for the Federal Circuit (CAFC) affirmed the earlier decision by the Patent Trial and Appeal Board (PTAB), which found all claims of the ‘793 Patent to be unpatentable due to the existence of prior art cited by Liquidia in inter partes review proceedings.

Additionally, in September 2023, UTHR filed a second complaint for patent infringement in District Court in Case No. 1:23-cv-00975-RGA (the New Hatch-Waxman Litigation). As of January 22, 2024, the only patent at issue is U.S. Patent No. 11,826,327 (the ‘327 Patent) which issued November 30, 2023. UTHR has stipulated to the dismissal of the ‘793 Patent from the New Hatch-Waxman Litigation as a result of the CAFC decision affirming invalidity of the '793 Patent. The ’327 Patent, the sole remaining patent at issue in the New Hatch-Waxman Litigation, was not issued before Liquidia submitted the NDA for Yutrepia in January 2020 to treat PAH. Therefore, the Company believes that final FDA approval for Yutrepia will not be subject to any statutory 30-month stay arising from the New Hatch-Waxman Litigation per Section 505(c)(3)(C) of the Federal Food, Drug and Cosmetic Act.

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

Read https://www.drugs.com/nda/yutrepia_240125.html

Liquidia Corporation Provides Update on New Drug Application for Yutrepia (treprostinil) inhalation powder

FDA Approves Eohilia (budesonide oral suspension) for the Treatment of Eosinophilic Esophagitis

 Takeda (TSE:4502/NYSE: TAK)   announced the U.S. Food and Drug Administration (FDA)   approval of Eohilia (budesonide oral suspension), the first and only FDA-approved oral therapy for people 11 years and older with eosinophilic esophagitis (EoE).1 It will be available in 2 mg/10 mL convenient, single-dose stick packs by the end of February.

Eohilia is a corticosteroid indicated for 12 weeks of treatment in patients 11 years and older with EoE.1 Developed specifically for EoE, Eohilia’s novel formulation of budesonide confers thixotropic properties – flowing more freely when shaken and returning to a more viscous state when swallowed.1,2

“Various formulations of corticosteroids have been used in the past to manage EoE, but in an off-label capacity and using multiple delivery options. With Eohilia, it’s gratifying to now have an FDA-approved treatment specifically formulated for a consistent dose delivery with demonstrated ability to address esophageal inflammation and EoE dysphagia symptoms,” said Ikuo Hirano, MD, professor of medicine and director of the Kenneth C. Griffin Esophageal Center in the Division of Gastroenterology and Hepatology at Northwestern University Feinberg School of Medicine. “As the treatment needs and goals of patients with EoE can vary, I welcome the flexibility that Eohilia offers as an oral medication.”

The FDA approval of Eohilia 2 mg twice daily is based on efficacy and safety data from two multicenter, randomized, double-blind, parallel-group, placebo-controlled 12-week studies (Study 1 and Study 2) in patients (ages 11 to 56 and 11 to 42, respectively) with EoE.1 In both studies, patients received at least one dose of either Eohilia 2 mg twice daily or placebo orally twice daily. Efficacy endpoints included histologic remission (peak eosinophil count of ≤6 per high-powered field across all available esophageal levels) and the absolute change from baseline in patient-reported Dysphagia Symptom Questionnaire (DSQ) combined score after 12 weeks of treatment. The DSQ measures how often a patient with EoE has trouble swallowing and the behavioral adaptations they subsequently use, as reported directly by patients.3

Significantly more patients receiving Eohilia achieved histologic remission vs. placebo in Study 1 (53.1% vs. 1%).1 In Study 2, 38% of Eohilia patients achieved histologic remission vs. 2.4% of those in the placebo group. Absolute change from baseline in DSQ combined score in the Eohilia vs. placebo groups in Study 1 was -10.2 (1.5) vs. -6.5 (1.8) and in Study 2, -14.5 (1.8) vs. -5.9 (2.1). During the last two weeks of each study, more patients receiving Eohilia experienced no dysphagia or only experienced dysphagia that “got better or cleared up on its own” as compared to placebo, as measured by the DSQ. Eohilia has not been shown to be safe and effective for the treatment of EoE for longer than 12 weeks. The most common adverse reactions (≥2% of patients receiving Eohilia and at a rate greater than placebo) in Study 1 included: respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%), headache (5%), gastroenteritis (3%), throat irritation (3%), adrenal suppression (2%) and erosive esophagitis (2%). The safety profile of Eohilia in Study 2 was generally similar to Study 1.1

“For most of us, eating is a simple experience. But for people living with eosinophilic esophagitis, sitting down for a meal can include painful and difficult swallowing, chest pain and a choking sensation,” said Brandon Monk, senior vice president and head, U.S. Gastroenterology Business Unit, Takeda. “With Eohilia, patients and their physicians now have the first and only FDA-approved oral treatment option for EoE that was shown during two 12-week clinical studies to reduce esophageal inflammation and improve the ability to swallow.”

EoE is a chronic, immune-mediated, inflammatory disease localized in the esophagus.4 Although the exact cause is unknown, it is believed to be triggered by a variety of stimuli including certain foods and environmental allergens.5,6 The chronic inflammation of EoE can lead to a range of symptoms, which can vary by person and age, and include difficulty swallowing, vomiting and pain.7 Identifying EoE can be complex and delayed diagnosis is common among patients. If left untreated, the inflammation of EoE can worsen and narrow the esophagus, which can lead to food impaction (when food becomes stuck in the esophagus).8,9 In fact, EoE is the leading cause of emergency room visits for food impaction.10

Takeda is assessing the financial impacts of the approval, including a reversal of impairment loss for intangible assets, on the fiscal year ending on March 31, 2024 (FY2023), but does not anticipate the impact to be material.

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

FDA Approves Eohilia (budesonide oral suspension) for the Treatment of Eosinophilic Esophagitis

FDA Approves Airsupra (albuterol/budesonide) Metered-Dose Inhaler to Reduce the Risk of Asthma Exacerbations

Airsupra (albuterol/budesonide), formerly known as PT027, has been approved in the US for the as-needed treatment or prevention of bronchoconstriction and to reduce the risk of exacerbations in people with asthma aged 18 years and older.

The approval by the Food and Drug Administration (FDA) was based on results from the MANDALA and DENALI Phase III trials. In MANDALA, Airsupra significantly reduced the risk of severe exacerbations compared to albuterol in patients with moderate to severe asthma when used as an as-needed rescue medication in response to symptoms. Importantly, in the secondary endpoint of mean annualised total systemic corticosteroid exposure, Airsupra demonstrated a significant reduction compared to albuterol at the approved dose of 180mcg albuterol/160mcg budesonide. In DENALI, Airsupra significantly improved lung function compared to the individual components albuterol and budesonide in patients with mild to moderate asthma.

\

Airsupra is a first-in-class, pressurised metered-dose inhaler (pMDI), fixed-dose combination rescue medication containing albuterol, a short-acting beta2-agonist (SABA), and budesonide, an anti-inflammatory inhaled corticosteroid (ICS) in the US. It is being developed by AstraZeneca and Avillion.

Bradley E. Chipps, Past President of the American College of Allergy, Asthma & Immunology and Medical Director of Capital Allergy & Respiratory Disease Center in Sacramento, US, said: “People with asthma are at risk of severe exacerbations regardless of their disease severity or level of control. Current albuterol rescue inhalers alleviate acute symptoms, but do not treat the underlying inflammation in asthma. The approval of Airsupra means that for the first time, adults with asthma in the US have a rescue treatment to manage both their symptoms and the inflammatory nature of their disease.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “With patients experiencing more than 10 million asthma exacerbations each year in the US and uncontrolled asthma expected to cost the US economy billions of dollars in direct medical costs alone over the next 20 years, today’s positive decision is good news for those adults with asthma who make up more than 80% of asthma patients in the US. Physicians will be able to offer their patients Airsupra, an important new rescue treatment that reduces the risk of asthma exacerbations.”

Asthma is a chronic, inflammatory respiratory disease with variable symptoms that affects as many as 262 million people worldwide.3 In the US over 21 million adults have asthma, representing more than 80% of the total number of people with asthma.4 Adults have 8.5 million exacerbations each year in the US.4 Uncontrolled asthma will cost the US economy an estimated $300 billion (in 2018 dollar values) in the next 20 years in direct medical costs alone.5

The safety and tolerability of Airsupra in both trials were consistent with the known profiles of the components,1,2 with the most common adverse events including headache, oral candidiasis, cough and dysphonia

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

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

FDA Approves Airsupra (albuterol/budesonide) Metered-Dose Inhaler to Reduce the Risk of Asthma Exacerbations

Scientists Develop Sensor That Tests Saliva for Breast Cancer

Mammograms are a lifesaving misery for middle-aged women, but a new tool could make getting a breast cancer screening as easy as spitting.

A new hand-held biosensor can detect breast cancer biomarkers from a tiny sample of saliva, researchers report Feb. 13 in the Journal of Vacuum Science & Technology B.

“Our device is an excellent choice because it is portable -- about the size of your hand -- and reusable,” said lead researcher Hsaio-Hsuan Wan, a doctoral student at the University of Florida. “The testing time is under five seconds per sample, which makes it highly efficient.”

The device uses paper test strips treated with specific antibodies that respond to targeted cancer biomarkers, the researchers explained.

When a saliva sample is placed on the strip, pulses of electricity are sent to contact points on the device.

These pulses cause the biomarkers to bind to the antibodies, which alters the electrode’s output signal enough to provide readings regarding cancer risk.

By comparison, mammograms, ultrasounds and MRI scans are all costly and require big pieces of equipment and low-dose radiation exposure, Wan said.

“In many places, especially in developing countries, advanced technologies like MRI for breast cancer testing may not be readily available,” she added in a university news release.

“Our technology is more cost-effective, with the test strip costing just a few cents and the reusable circuit board priced at $5,” Wan added. “We are excited about the potential to make a significant impact in areas where people might not have had the resources for breast cancer screening tests before.”

The device can provide accurate test results with just a drop of saliva, even if the concentration of the cancer biomarker is a minuscule one-quadrillionth of a gram per milliliter.

One of the biomarkers involves human epidermal growth factor receptor 2 (HER2), a protein that drives 15% to 20% of invasive breast cancers. Another is CA 15-3, an antigen released into the bloodstream by breast cancer.

Results showed the device could distinguish between healthy breast tissue, early breast cancer and advanced breast cancer, using those two biomarkers. Researchers tested the device in 21 human saliva samples.

“The highlight for me was when I saw readings that clearly distinguished between healthy individuals and those with cancer,” Wan said. “We dedicated a lot of time and effort to perfecting the strip, board and other components. Ultimately, we’ve created a technique that has the potential to help people all around the world.”

Ref: https://www.eurekalert.org/news-releases/1033951

Ref: https://pubs.aip.org/avs/jvb/article/42/2/023202/3262988/High-sensitivity-saliva-based-biosensor-in

Scientists Develop Sensor That Tests Saliva for Breast Cancer  

Plant-Based Food Intake Linked to Better QoL in Prostate Cancer

Among patients with prostate cancer, greater consumption of plant-based foods is associated with higher scores in quality-of-life domains, according to a study published online Feb. 13 in Cancer.

Stacy Loeb, M.D., Ph.D., from New York University and Manhattan Veterans Affairs in New York City, and colleagues examined the relationship between plant-based diet indices after prostate cancer diagnosis and quality of life in a prospective cohort study involving 3,505 participants in the Health Professionals Follow‐Up Study (1986 to 2016) with nonmetastatic prostate cancer. Overall and healthful plant-based diet indices were calculated using food-frequency questionnaires. The Expanded Prostate Cancer Index Composite was used to calculate quality-of-life scores.

The researchers found that better scores for sexual function, urinary irritation/obstruction, urinary incontinence, and hormonal/vitality were seen in association with a higher plant-based diet index. In the age-adjusted analysis, but not in the multivariable analysis, consuming more healthful plant-based foods was also associated with better sexual and bowel function and improved urinary incontinence and hormonal/vitality scores.

"Individuals with prostate cancer should be advised that incorporating a greater amount of plant‐based foods into their diet could not only reduce the risk of comorbid conditions but also contribute to improved functional outcomes," the authors write.

Ref : https://acsjournals.onlinelibrary.wiley.com/doi/abs/10.1002/cncr.35172

Plant-Based Food Intake Linked to Better QoL in Prostate Cancer - Drugs.com MedNews

FDA Approves Zepbound (tirzepatide) for Chronic Weight Management

• 
• The U.S. Food and Drug Administration (FDA) approved Eli Lilly and Company's (NYSE: LLY) Zepbound™ (tirzepatide) injection, the first and only obesity treatment of its kind that activates both GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1) hormone receptors. Zepbound is indicated for adults with obesity (with a BMI of 30 kg/m2 or greater), or those who are overweight (with a BMI of 27 kg/m2 or greater) and also have weight-related medical problems such as hypertension, dyslipidemia, type 2 diabetes mellitus, obstructive sleep apnea or cardiovascular disease, to lose weight and keep it off. It should be used with a reduced-calorie diet and increased physical activity. Zepbound should not be used with other tirzepatide-containing products or any GLP-1 receptor agonist medicines, and it has not been studied in patients with a history of pancreatitis, or with severe gastrointestinal disease, including severe gastroparesis.
• "Obesity is a chronic disease that can result in serious health complications, including heart disease, stroke and diabetes. Despite our knowledge of obesity as a treatable, chronic disease, people living with obesity still face many challenges in their health and weight management journey," said Joe Nadglowski, president and chief executive officer of the Obesity Action Coalition. "New treatment options bring hope to the many people with obesity who struggle with this disease and are seeking better options for weight management."
• The approval was based on results from the phase 3 SURMOUNT-1 and SURMOUNT-2 trials. In SURMOUNT-1, a study in 2,539 adults with obesity, or excess weight and weight-related medical problems not including diabetes, people taking Zepbound as an adjunct to diet and exercise experienced substantial weight loss compared with placebo at 72 weeks. At the highest dose (15 mg), people taking Zepbound lost on average 48 lb., while at the lowest dose (5 mg), people lost on average 34 lb. (compared to 7 lb. on placebo).
• Additionally, 1 in 3 patients taking Zepbound at the highest dose lost over 58 lb. (25% of body weight), compared to 1.5% on placebo, according to data not controlled for type 1 error. The average starting weight was 231 lb.
• While not approved to treat these conditions, in a clinical trial, people who dieted, exercised and took Zepbound for the treatment of obesity or overweight with weight-related medical problems observed changes in cholesterol and reductions in blood pressure and waist size.
• "Unfortunately, despite scientific evidence to the contrary, obesity is often seen as a lifestyle choice – something that people should manage themselves," said Dr. Leonard Glass, senior vice president global medical affairs, Lilly Diabetes and Obesity. "For decades, diet and exercise have been a go-to, but it's not uncommon for a person to have tried 20-30 times to lose weight with this approach. Research now shows that the body may respond to a calorie-deficit diet by increasing hunger and reducing feelings of fullness, making weight loss more difficult. Lilly is aiming to eliminate misperceptions about this disease and transform how it can be managed."
• Zepbound use may be associated with gastrointestinal adverse reactions, sometimes severe. The most commonly reported adverse events (observed in ≥ 5% of clinical trial participants) were nausea, diarrhea, vomiting, constipation, abdominal pain, dyspepsia, injection-site reactions, fatigue, hypersensitivity reactions, eructation, hair loss and gastroesophageal reflux disease.1 In studies, most nausea, diarrhea and vomiting occurred when people increased their dose – but the effects generally decreased over time. In studies, gastrointestinal side effects were more common in people taking Zepbound than people taking placebo, and people taking Zepbound were more likely than those on placebo to stop treatment because of these side effects. The label for Zepbound includes a Boxed Warning regarding thyroid C-cell tumors. Zepbound is contraindicated in patients with a personal or family history of medullary thyroid carcinoma, in patients with Multiple Endocrine Neoplasia syndrome type 2, and in patients with known serious hypersensitivity to tirzepatide or any of the excipients in Zepbound. See Important Safety Information below and full Prescribing Information and Medication Guide.
• "Far too many hurdles continue to prevent people living with obesity from accessing obesity treatments that could lead to significant weight loss," said Mike Mason, executive vice president and president, Lilly Diabetes and Obesity. "Broader access to these medicines is critical, which is why Lilly is committed to working with healthcare, government and industry partners to ensure people who may benefit from Zepbound can access it."
• Zepbound is expected to be available in the U.S. by the end of the year in six doses (2.5 mg, 5 mg, 7.5 mg, 10 mg, 12.5 mg, 15 mg) at a list price of $1,059.87, which is approximately 20% lower than semaglutide 2.4 mg injection for weight loss. List price does not reflect the typical out-of-pocket cost to patients given insurance coverage and discounts. Lilly is putting a commercial savings card program in place that will help people who may benefit from Zepbound better access it.

• People who are commercially insured with coverage for Zepbound may be eligible to pay as low as $25 for a 1-month or 3-month prescription.
• People who are commercially insured without coverage for Zepbound may be eligible to pay as low as $550 for a 1-month prescription of Zepbound, approximately 50% lower than the list price.
• People may begin using the savings card program in the days following product availability at U.S. pharmacies. To learn more about these programs, or to sign up to receive the latest news, please visit www.Zepbound.lilly.com. Terms and conditions apply.
• Tirzepatide is also under regulatory review for weight management in Europe, China, the United Kingdom and several additional markets.

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FDA Approves Zepbound (tirzepatide) for Chronic Weight Management

FDA Approves DefenCath (taurolidine and heparin) to Reduce the Incidence of Catheter-Related Bloodstream Infections in Adult Hemodialysis Patients

CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening diseases and conditions, announced  the U.S. Food and Drug Administration (FDA)  approval of  DefenCath® (taurolidine and heparin) catheter lock solution (CLS) to reduce the incidence of catheter-related bloodstream infections (CRBSIs) for the limited population of adult patients with kidney failure receiving chronic hemodialysis through a central venous catheter (CVC). DefenCath is the first and only FDA-approved antimicrobial CLS in the U.S. and was shown to reduce the risk of CRBSIs by up to 71% in a Phase 3 clinical study.

Joseph Todisco, Chief Executive Officer of CorMedix commented, “The approval of DefenCath marks a major advancement in reducing life-threatening infections for patients receiving hemodialysis via central venous catheters and an important milestone for CorMedix. As the first FDA-approved antimicrobial catheter lock solution designed to prevent CRBSIs, DefenCath offers healthcare providers an option to reduce the risk of infections in a patient population already vulnerable due to underlying kidney failure. We thank all the patients, caregivers, clinical investigators, and our employees who have played an integral role in the development and regulatory approval of DefenCath. Our commercial team along with our broader organization is preparing for commercial launch, and we look forward to working with healthcare providers and facilities to give hemodialysis patients access to DefenCath in early 2024.”

The FDA approval of DefenCath was supported by results from the randomized, double-blind, active control, multicenter pivotal Phase 3 LOCK-IT-100 clinical trial designed to assess the efficacy and safety of DefenCath for reducing the incidence of CRBSIs in patients with kidney failure receiving chronic hemodialysis. In the study, a total of 806 subjects were randomized to receive either DefenCath or heparin as a CLS. Patients in the DefenCath group had a lower incidence of CRBSI events compared to patients in the control group. The Hazard Ratio was 0.29, corresponding to a statistically significant 71% reduction in risk of developing a CRBSI. An independent Data Safety and Monitoring Board recommended an early termination of the study based on demonstrated efficacy and a pre-specified level of statistical significance with no safety concerns. Adverse events were comparable to control.

Edward V. Hickey, III, President of the American Association of Kidney Patients and Chair of the Veterans Health Initiative stated, “Patients and their loved ones have faced many burdens related to kidney failure, including complications caused by catheter related bloodstream infections and associated loss of work, severe disability and death. Until now, patients who need hemodialysis via a central venous catheter have had little choice other than to accept high infection risks associated with the existing standard of care. The FDA’s approval of DefenCath is a meaningful moment for patients and their healthcare providers because they now have a new alternative to reduce the risks of CRBSIs.” Mr. Hickey is a kidney patient, former senior staff member of the U.S. Congress and has served in two presidential administrations.

More at...

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

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

FDA Approves DefenCath (taurolidine and heparin) to Reduce the Incidence of Catheter-Related Bloodstream Infections in Adult Hemodialysis Patients

FDA Approves Truqap (capivasertib) plus Faslodex for Patients with Advanced HR-Positive Breast Cancer

AstraZeneca’s Truqap (capivasertib) in combination with Faslodex (fulvestrant) has been approved in the US for the treatment of adult patients with hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1 or PTEN). Eligible patients will have progressed on at least one endocrine-based regimen in the metastatic setting or experienced recurrence on or within 12 months of completing adjuvant therapy.

The approval by the Food and Drug Administration (FDA) was based on the results from the CAPItello-291 Phase III trial published earlier this year in The New England Journal of Medicine.1 In the trial, Truqap in combination with Faslodex reduced the risk of disease progression or death by 50% versus Faslodex alone in patients with tumours harbouring PI3K/AKT pathway biomarker alterations (based on hazard ratio of 0.50, 95% confidence interval 0.38-0.65; p=<0.001; median progression-free survival (PFS) 7.3 versus 3.1 months).

Breast cancer is the most common cancer and one of the leading causes of cancer-related death worldwide.2 HR-positive breast cancer (expressing estrogen or progesterone receptors, or both), is the most common subtype, with more than 65% of tumours considered HR-positive and HER2-low or HER2-negative.3 Collectively, mutations in PIK3CA, AKT1 and alterations in PTEN occur frequently, affecting up to 50% of patients with advanced HR-positive breast cancer.4-6 Endocrine therapies are widely used in this setting, but many patients develop resistance to 1st-line cyclin-dependent kinase (CDK) 4/6 inhibitors and estrogen receptor-targeting therapies, underscoring the need for additional endocrine therapy-based options

Ref; https://en.wikipedia.org/wiki/Capivasertib

FDA Approves Truqap (capivasertib) plus Faslodex for Patients with Advanced HR-Positive Breast Cancer

FDA Approves Ogsiveo (nirogacestat) for Adults with Desmoid Tumors

SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced  the U.S. Food and Drug Administration (FDA) approval of  Ogsiveo™ (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. The FDA previously granted breakthrough therapy, fast track and orphan drug designations to nirogacestat for the treatment of desmoid tumors.

“Our team is honored to deliver the first FDA-approved therapy for patients with desmoid tumors. This community has been waiting for an effective treatment that not only shrinks their tumors but also significantly improves pain, which is the most debilitating symptom reported by people living with desmoid tumors,” said Saqib Islam, Chief Executive Officer of SpringWorks. "We are pleased with the broad label, which includes all progressing adult patients and specifically references improvement in pain, and believe Ogsiveo has the potential to become the new standard of care for people living with these devastating tumors. This is a watershed moment for the desmoid tumor community, and we would like to extend our gratitude to the patients, families, investigators, and advocacy groups involved in the journey to making Ogsiveo available in the U.S.”

Desmoid tumors are locally aggressive and invasive soft-tissue tumors that can lead to substantial morbidity.2,3 In addition, when vital structures are impacted, desmoid tumors can be life-threatening.3 Although they do not metastasize, desmoid tumors are often refractory to existing off-label systemic therapies and associated with recurrence rates of up to 77% following surgical resection.4-6 Desmoid tumor experts and treatment guidelines now recommend systemic therapies as first-line intervention instead of surgery for most tumor locations requiring treatment.6

“Desmoid tumors can have a significant impact on people’s lives and are difficult to manage due to their invasive nature and high rates of recurrence. Ogsiveo is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumor symptoms,” said Mrinal M. Gounder, M.D., sarcoma medical oncologist at Memorial Sloan Kettering Cancer Center (MSK) in New York City and an investigator in the Phase 3 DeFi trial. “As a treating physician, it was encouraging to see in the DeFi trial that Ogsiveo achieved statistically significant and clinically meaningful improvements across the primary and all key secondary endpoints, while also having a manageable safety profile. This approval represents an important therapeutic advance for patients.”

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

FDA Approves Ogsiveo (nirogacestat) for Adults with Desmoid Tumors

FDA Approves Fabhalta (iptacopan) for the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Novartis announced the U.S. Food and Drug Administration (FDA) approval of  Fabhalta® (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)1. Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]). In clinical trials, treatment with Fabhalta increased hemoglobin levels (≥ 2 g/dL from baseline in the absence of RBC transfusions) in the majority of patients and in APPLY-PNH nearly all patients treated with Fabhalta did not receive blood transfusions.

“An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with PNH,” said Vinod Pullarkat, MD, MRCP, Clinical Professor, Department of Hematology and Hematopoietic Cell Transplantation, City of Hope. “In clinical studies, iptacopan was superior to anti-C5s in hemoglobin improvement in the absence of RBC transfusion and transfusion avoidance rate, and also effective in complement inhibitor-naïve individuals, by providing clinically meaningful hemoglobin-level increases without the need for blood transfusions.”

The FDA approval is based on the Phase III APPLY-PNH trial in patients with residual anemia (hemoglobin < 10 g/dL) despite prior anti-C5 treatment who switched to Fabhalta, which demonstrated superiority in hemoglobin improvement in the absence of RBC transfusions and in transfusion avoidance rate over patients who stayed on anti-C5 treatments1,2. Approval was also supported by the Phase III APPOINT-PNH study in complement inhibitor-naïve patients1,3. The 24-week core treatment periods in APPLY-PNH and APPOINT-PNH trials respectively showed

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

FDA Approves Fabhalta (iptacopan) for the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

FDA Approves iDose TR (travoprost intracameral implant) for the Treatment of Glaucoma

Glaukos Corporation (NYSE: GKOS), an ophthalmic medical technology and pharmaceutical company focused on novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases, announced   the U.S. Food and Drug Administration (FDA) approval of its New Drug Application (NDA) for a single administration per eye of iDose® TR (travoprost intracameral implant) 75 mcg, a prostaglandin analog indicated for the reduction of intraocular pressure (IOP) in patients with ocular hypertension (OHT) or open-angle glaucoma (OAG).

“With the next generation of procedural pharmaceutical solutions for glaucoma such as iDose TR, we now have a new tool that will confront the standard legacy practice of relying on topical drops, which are known to cause uncomfortable side effects and present a myriad of challenges such as treatment adherence, complex dosing regimens, and difficulty with self-administration,” said John Berdahl, MD, clinician and researcher at Vance Thompson Vision. “The clinical data suggest that iDoseTR is not only effective with a favorable safety profile, but it has potential to relieve patients from the burdens of prescription eye drops for an extended period of time. I look forward to adding this novel therapy into my treatment toolbox for the benefit of my patients.”

The FDA approval is based on results from two prospective, randomized, multicenter, double-masked, Phase 3 pivotal trials (GC-010 and GC-012) designed to compare the safety and efficacy of a single administration of one of two iDose TR models with different travoprost release rates (referred to as the fast- and slow-release iDose TR models, respectively) to topical timolol ophthalmic solution, 0.5% BID (twice a day), in reducing IOP in subjects with open-angle glaucoma or ocular hypertension. In total, the Phase 3 trials randomized 1,150 subjects across 89 clinical sites. The FDA approval and Phase 3 data referenced below is for the slow-release iDose TR model, consistent with the company’s NDA submission and commercialization plans.

Both Phase 3 trials successfully achieved the pre-specified primary efficacy endpoints through 3 months and demonstrated a favorable tolerability and safety profile through 12 months. IOP reductions from baseline over the first 3 months were 6.6-8.4 mmHg in the iDose TR arm, versus 6.5-7.7 mmHg in the timolol control arm (mmHg range represents IOP reduction means across the six U.S. FDA pre-specified timepoints of 8 a.m. and 10 a.m. at Day 10, Week 6 and Month 3). Based on these outcomes, the FDA concluded in the prescribing information that iDose TR demonstrated non-inferiority to timolol ophthalmic solution in IOP reduction during the first 3 months. The FDA also noted that subsequently iDose TR did not demonstrate non-inferiority over the next 9 months.

iDose TR is a first-of-its-kind, long-duration, intracameral procedural pharmaceutical therapy designed to continuously deliver 24/7 therapeutic levels of a proprietary formulation of travoprost inside the eye for extended periods of time. iDose TR is intended to improve the standard of care by addressing the ubiquitous patient non-compliance issues and chronic side effects associated with topical glaucoma medications.

“The FDA approval of iDose TR represents a significant milestone for Glaukos following an extensive pioneering journey since the inception of the original idea nearly 15 years ago. Today’s approval ushers in a new era of interventional glaucoma therapy by enabling a more proactive and reliable approach for patients in need,” said Thomas Burns, Glaukos chairman and chief executive officer. “We believe iDose TR can be a transformative, novel technology able to fundamentally improve the treatment paradigm for patients with open-angle glaucoma or ocular hypertension. We are grateful to the clinical investigators and study participants in the clinical trials for their instrumental roles in helping us reach this important advancement for glaucoma patient care. At Glaukos, we are relentlessly focused on delivering novel therapies for chronic eye diseases and now iDoseTR has the potential to redefine the standard of care for patients in the U.S. affected by open-angle glaucoma and ocular hypertension.”

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

FDA Approves iDose TR (travoprost intracameral implant) for the Treatment of Glaucoma

FDA Approves Iwilfin (eflornithine) as Maintenance Therapy for High-Risk Neuroblastoma

USWM, LLC  announced the U.S. Food and Drug Administration (FDA)  approval of Iwilfin ™ (eflornithine) 192 mg tablets, a groundbreaking oral maintenance therapy for high-risk neuroblastoma. Iwilfin is indicated to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.

Acc

ording to the American Cancer Society, 700-800 cases of neuroblastoma are diagnosed in the U.S. each year, with 90% of diagnoses coming before age 5. Over 50% of these cases are classified as high-risk. High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission. Approximately half of children with high-risk neuroblastoma do not survive beyond five years from diagnosis. Although existing treatments are effective in helping patients achieve remission, patients lack options to sustain it. Avoiding relapse is crucial to improving survival rates.

“We are thrilled to announce the FDA approval of Iwilfin , which provides a new and much-needed treatment option for children with high-risk neuroblastoma,” said Breck Jones, Chief Executive Officer of US WorldMeds. “The goal for treating these young patients is to prevent relapse, and advancing therapeutic options is critical to this mission. Iwilfin offers new hope and improved outcomes for these vulnerable children.”

Iwilfin is taken orally, with or without food, twice daily for two years. Iwilfin is generally well-tolerated, with side effects typically manageable through dose modifications. The most common side effects are hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. Important Safety Information can be found below.

US WorldMeds partnered with the Beat Childhood Cancer Research Consortium at Penn State University, which conducted the preclinical and clinical research to help advance this vital therapy. The Consortium represents a group of over 50 hospitals that offer collaboration through a network of childhood cancer clinical trials.

“Our partnerships were instrumental in bringing Iwilfin through the FDA registration process,” said Kristen Gullo, Vice President of Development and Regulatory Affairs at US WorldMeds. “We are thankful for the dedication of our partners, specifically the Beat Childhood Cancer Research Consortium, who work tirelessly to improve treatment outcomes for pediatric cancer patients. This FDA approval represents a beacon of hope for the high-risk neuroblastoma community and a significant step forward in the fight against this devastating disease.”

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

FDA Approves Iwilfin (eflornithine) as Maintenance Therapy for High-Risk Neuroblastoma

FDA Approves Wainua (eplontersen) for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced  the U.S. Food and Drug Administration (FDA)   approval of  Ionis and AstraZeneca's Wainua™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.

"Many people living with hereditary transthyretin-mediated amyloid polyneuropathy are unable to fully enjoy their lives because of the relentless, progressive and debilitating  effects of the disease," said Michael J. Polydefkis, M.D., professor of neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study. "Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease."

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. Wainua is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source.

"The FDA approval of Wainua marks an important milestone for people living with hereditary transthyretin-mediated amyloid polyneuropathy, who will now have an effective, well-tolerated treatment that can be self-administered via auto-injector to combat this devastating disease," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "It is also a pivotal moment for Ionis as Wainua will be the first in a steady cadence of potential commercial launches for the company. We are proud to have discovered and, together with AstraZeneca, developed Wainua, and are grateful to the patients, caregivers and investigators who participated in our clinical studies, as well as for the dedication of our scientists and researchers."

"People with hereditary transthyretin-mediated amyloid polyneuropathy, and other forms of amyloidosis, are often misdiagnosed since symptoms can mirror other conditions," said Isabelle Lousada, President and CEO, Amyloidosis Research Consortium "The path to getting an accurate diagnosis can often be a long, arduous journey and it is critical that a timely and accurate diagnosis is made not only for the individual experiencing symptoms but for their families and loved ones. It is exciting to see new innovations coming through and increased efforts to raise awareness in an area that has often been overlooked or neglected."

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

Ref : https://go.drugbank.com/drugs/DB16199

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FDA Approves Wainua (eplontersen) for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis