NDA

Santhera Announces NDA for Vamorolone in Duchenne Muscular Dystrophy Accepted and Granted Priority Review by China’s NMPA

Retrieved on: 
Wednesday, March 27, 2024
Diagnosis, NMPA, Patient, Ageing, NDA, Breakthrough, CDE, Duchenne muscular dystrophy, Therapy, Vamorolone, Safety, File, DMD, Neuromuscular disease, Pharmaceutical industry

Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China.

Key Points: 
  • Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China.
  • The Center for Drug Evaluation (CDE) of the Chinese drug authority NMPA accepted the filing and granted priority review for vamorolone in DMD for patients aged 4 years and older which could, subject to a positive outcome, lead to approval by Q1 2025.
  • Duchenne muscular dystrophy is a rare neuromuscular disease affecting about 70,000 patients in China.
  • The submission is further supported by a study which investigated the pharmacokinetic parameters of vamorolone in healthy adult Chinese volunteers.

AXSOME ALERT: Bragar Eagel & Squire, P.C. is Investigating Axsome Therapeutics, Inc. on Behalf of Long-Term Stockholders and Encourages Investors to Contact the Firm

Retrieved on: 
Tuesday, March 26, 2024
Symantec Endpoint Protection, Telephone, CRL, News, NDA, CMC, Migraine, Myenteric plexus, Chemistry, P.C, Therapy, FDA, Food, Pharmaceutical industry

Our investigation concerns whether the board of directors of Axsome have breached their fiduciary duties to the company.

Key Points: 
  • Our investigation concerns whether the board of directors of Axsome have breached their fiduciary duties to the company.
  • Axsome is a biopharmaceutical company that engages in the development of novel therapies for central nervous system disorders in the United States.
  • However, unbeknownst to investors, the Company’s preparation and eventual submission of the AXS-07 NDA was plagued with chemistry, manufacturing, and control (“CMC”) issues.
  • Then, on April 25, 2022, Axsome disclosed in a filing with the U.S. Securities and Exchange Commission that, “[o]n April 22, 2022, Axsome .

Shorla Oncology Announces FDA Filing Acceptance of New Drug Application to Treat Certain Forms of Leukemia and Other Cancers

Retrieved on: 
Monday, April 8, 2024
FDA, Pharmaceutical, Health, Oncology, Acute lymphoblastic leukemia, Growth, Disease, Caregiver, New Drug Application, Patient, Nelarabine, NDA, Breast, Leukemia, Agency, Cancer, T-cell leukemia, Adenocarcinoma, Ovary, Food, Pharmaceutical industry

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) for SH-201, the first palatable oral liquid of the related chemotherapeutic agent to treat certain forms of leukemia and other cancers.

Key Points: 
  • Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) for SH-201, the first palatable oral liquid of the related chemotherapeutic agent to treat certain forms of leukemia and other cancers.
  • The Agency assigned a Prescription Drug User Fee Act (“PDUFA”) action date of November 30, 2024.
  • “With this NDA acceptance for SH-201, we are a step closer to providing an alternative treatment to thousands of U.S. patients diagnosed with leukemia and other cancers who do not currently have the option of an oral liquid,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology.
  • The company recently raised $35 million in Series B funding that has allowed Shorla to accelerate the growth of its oncology portfolio.

FDA Accepts Filing of New Drug Application for Nalmefene Auto-injector for the Treatment of Known or Suspected Opioid Overdose

Retrieved on: 
Monday, April 8, 2024
General Health, FDA, Health, Pharmaceutical, Medical Supplies, Hypoventilation, Hypersensitivity, Nalmefene, Pulmonary edema, Caregiver, Safety, Tachycardia, Risk, New Drug Application, Patient, Ventricular tachycardia, Naloxone, Health care, Buprenorphine, Mortality, NDA, MD, Methadone, Ventricular fibrillation, Hypotension, Oxygen, LAAM, Recurrence, Allergy, Purdue University, Hypertension, Fentanyl, Pharmaceutical industry

Purdue Pharma L.P. (“Purdue”) announced today that FDA has accepted for filing the company’s New Drug Application (NDA) for the auto-injector delivery form of nalmefene hydrochloride injection*.

Key Points: 
  • Purdue Pharma L.P. (“Purdue”) announced today that FDA has accepted for filing the company’s New Drug Application (NDA) for the auto-injector delivery form of nalmefene hydrochloride injection*.
  • "Through this auto-injector FDA submission, our goal is to help expand the availability of nalmefene to the community alongside existing available options for healthcare professionals.
  • Priority review status means that FDA will expedite the review process to evaluate a drug that would significantly improve treatment of a serious condition.
  • Nalmefene Hydrochloride Injection is indicated in the management of known or suspected opioid overdose.

Alzheon Announces that United States Federal Circuit Affirmed Decision of U.S. Patent Office Invalidating Risen (Suzhou) Pharmaceutical Technology Co., Ltd.’s Patent on Isotopically Enriched Forms of ALZ-801/Valiltramiprosate

Retrieved on: 
Monday, April 8, 2024
Biotechnology, Professional Services, Health, Patent Law, Pharmaceutical, Neurology, Research, Genetics, Legal, Science, Clinical Trials, Comment, Patent, Patient, NDA, PTAB, Federal Circuit Court, Doctor of Philosophy, IPR, Prodrug, Rule 34, Pharmaceutical industry

The decision was affirmed without discussion by the Federal Circuit Court (Case No.

Key Points: 
  • The decision was affirmed without discussion by the Federal Circuit Court (Case No.
  • In 2020, Alzheon petitioned the Patent Trial and Appeal Board (PTAB) of the U.S. Patent Office (USPTO) to institute an Inter-Parties Review (IPR) of Risen’s U.S. Patent No.
  • Risen appealed the PTAB’s decision to the United States Court of Appeals for the Federal Circuit.
  • “Alzheon is pleased that the U.S. Federal Circuit affirmed the Patent Trial and Appeal Board’s decision to invalidate all claims of Risen’s patent.

STEALTH BIOTHERAPEUTICS ANNOUNCES FDA ACCEPTANCE OF NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME

Retrieved on: 
Monday, April 8, 2024
Friends, New Drug Application, Elamipretide, Patient, Barth syndrome, NDA, Syndrome, Ultra, Quality of life, Safety, Muscle weakness, Stealth, Fatigue, FDA, Food, Medicine, Pharmaceutical industry

NEEDHAM, Mass., April 8, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has accepted for filing its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome. The NDA is supported by the positive data from the SPIBA-001 Phase 3 Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020. 

Key Points: 
  • Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020.
  • The FDA indicated that it is currently planning to hold an advisory committee meeting to discuss the application.
  • The application was assigned a standard review designation, which the Company has asked FDA to reconsider.
  • Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024.

FDA Grants Breakthrough Therapy Designation to Sunvozertinib for the First-Line Treatment of Patients with advanced Non-Small Cell Lung Cancer Harboring EGFR Exon 20 Insertion Mutations

Retrieved on: 
Sunday, April 7, 2024
PART, EGFR, Mutation, BTD, NSCLC, Disease, Progression-free survival, Patient, Heel, Corr, NDA, Breakthrough, Non-Euclidean geometry, Doctor of Philosophy, CDE, Society, ESMO, Safety, ASCO, FDA, Annual general meeting, Food, Pharmaceutical industry

SHANGHAI, April 7, 2024 /PRNewswire/ -- Dizal (688192.SH) today announced that the U.S. Food and Drug Administration ("FDA") has granted Breakthrough Therapy Designation (BTD) to its sunvozertinib as the first-line treatment for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion (Exon20ins) mutations.

Key Points: 
  • SHANGHAI, April 7, 2024 /PRNewswire/ -- Dizal (688192.SH) today announced that the U.S. Food and Drug Administration ("FDA") has granted Breakthrough Therapy Designation (BTD) to its sunvozertinib as the first-line treatment for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion (Exon20ins) mutations.
  • This Breakthrough Therapy Designation (BTD) approval was based on results from the global multi-center phase I/II study (WU-KONG1).
  • This new BTD will enable us work more closely with the FDA and accelerate its clinical development and regulatory submission."
  • Affecting roughly 2%-4% of NSCLC patients, EGFR Exon20ins mutations have been difficult to treat due to their unique spatial conformation, diverse mutation subtypes, and high heterogeneity.

Belite Bio Announces PMDA Submission of Tinlarebant for Stargardt Disease Clinical Trial in Japan

Retrieved on: 
Friday, March 22, 2024
Retina, Medication, SAN, NDA, Adolescence, Clinical trial, Safety, Pharmacokinetics, PMDA, Therapy, DRAGON, Pharmaceutical industry

In the Phase 2 trial, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment period

Key Points: 
  • In the Phase 2 trial, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment period
    SAN DIEGO, March 22, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announces its submission to the Pharmaceuticals and Medical Devices Agency (PMDA) to initiate a clinical trial of Tinlarebant in adolescent STGD1 in Japan (“DRAGON II”).
  • The DRAGON II trial is a combination of Phase 1b open-label study to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent STGD1 subjects and a Phase 2/3, global, multicenter, double-masked, placebo-controlled, randomized study designed to evaluate the efficacy, safety and tolerability of Tinlarebant in adolescent STGD1 subjects.
  • Approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects, are targeted for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization (tinlarebant:placebo).
  • The data from Japanese subjects is intended to facilitate future NDA applications in Japan.

Journey Medical Corporation Reports Full-Year 2023 Financial Results and Recent Corporate Highlights

Retrieved on: 
Thursday, March 21, 2024
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SCOTTSDALE, Ariz., March 21, 2024 (GLOBE NEWSWIRE) -- Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical” or “the Company”), a commercial-stage pharmaceutical company that primarily focuses on the selling and marketing of U.S. Food and Drug Administration (“FDA”)-approved prescription pharmaceutical products for the treatment of dermatological conditions, today announced financial results and recent corporate highlights for the full year ended December 31, 2023.

Key Points: 
  • ET to discuss the financial results and provide a business update
    SCOTTSDALE, Ariz., March 21, 2024 (GLOBE NEWSWIRE) -- Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical” or “the Company”), a commercial-stage pharmaceutical company that primarily focuses on the selling and marketing of U.S. Food and Drug Administration (“FDA”)-approved prescription pharmaceutical products for the treatment of dermatological conditions, today announced financial results and recent corporate highlights for the full year ended December 31, 2023.
  • Claude Maraoui, Journey Medical’s Co-Founder, President and Chief Executive Officer, said, “2023 was a year of growth and development for Journey Medical.
  • In December 2023, Journey Medical entered into a $20.0 million credit facility with SWK Holdings Corporation (“SWK”), a specialized finance company with a focus on the global healthcare sector.
  • In July 2023, Journey Medical announced positive topline results from the two DFD-29 Phase 3 clinical trials (MVOR-1 & MVOR-2) for the treatment of rosacea.

ARS Pharmaceuticals Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, March 21, 2024
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SAN DIEGO, March 21, 2024 (GLOBE NEWSWIRE) -- ARS Pharmaceuticals, Inc. (Nasdaq: SPRY), a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect patients from severe allergic reactions that could lead to anaphylaxis, today reported business updates and financial results for the fourth quarter and full year 2023.

Key Points: 
  • In February 2024, ARS Pharma announced the successful completion of the repeat dosing study of neffy in seasonal allergic rhinitis under nasal allergen challenge conditions.
  • R&D Expenses: Research and development expenses were $3.4 million and $20.3 million for the quarter and year ended December 31, 2023, respectively.
  • G&A Expenses: General and administrative expenses were $6.8 million and $47.3 million for the quarter and year ended December 31, 2023, respectively.
  • Net Loss: Net loss was $7.2 million and $54.4 million for the quarter and year ended December 31, 2023, respectively.