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Stealth faces another hurdle as FDA delays decision on Barth syndrome drug
The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.
There’s an inconvenient fact in the new trans data
New research from the University of York has found that fifty times more children under 18 are now diagnosed with gender ...
Taiwan News2d
X4 Pharmaceuticals Announces EMA Validation of Marketing Authorization Application (MAA) for Mavorixafor for the Treatment of WHIM Syndrome
If approved by the EMA, mavorixafor would be the first drug indicated for patients with WHIM syndrome in Europe, a population estimated to be approximately 1,000 people. Earlier this month X4 ...
Malawi Nyasa Times3d
FDA authorizes marketing of 20 ZYN nicotine pouch products after extensive scientific review
Malawi breaking news publishing 24 hours a day news about Malawi, Malawi Business, Malawi Tourism, Malawi Politics, Malawi News ...
Monthly Prescribing Reference3d
FDA Extends Review of Elamipretide for Barth Syndrome
The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome, a rare genetic disorder.
FDA and EMA review Biogen's higher dose SMA drug
CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB), a $20.8 billion biotechnology company with a robust 75.6% gross profit margin, ...
Replimune Announces Biologics License Application Acceptance and Priority Review for RP1 for the Treatment of Advanced Melanoma
The confirmatory IGNYTE-3 trial is assessing RP1 in combination with nivolumab in patients with advanced melanoma who have progressed on anti-PD-1 and anti-CTLA-4 therapies or are ineligible for ...
JD Supra5d
Drugs and Biologics at FDA: Reflections on 2024 Activity Shed Light on the Road Ahead
With each new year comes a new set of accompanying challenges, from tackling the latest innovations to new incarnations of old issues. 2025 will be ...
The American Journal of Managed Care5d
In a First-of-Its-Kind Decision, FDA Will Allow Marketing of Nicotine Pouches
This marketing decision from the FDA covers 2 nicotine strengths, 3 mg and 6 mg, and 10 flavors of the small tobacco-free ...
Business Wire8d
Arrowhead Pharmaceuticals Announces Acceptance of New Drug Application by U.S. FDA of Plozasiran for the Treatment of Familial Chylomicronemia Syndrome
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has accepted ...
FDA Accepts Arrowhead Pharmaceuticals' Drug Application for Genetic Disease Treatment
Arrowhead Pharmaceuticals said Friday that the Food and Drug Administration accepted the company's new drug application for its treatment of familial chylomicronemia syndrome, a severe and rare ...
Monthly Prescribing Reference9d
Prefilled Syringe Presentation of Shingrix Under FDA Review
The new prefilled syringe has the same composition but removes the need for reconstitution, providing a more convenient option for health care providers who administer vaccines. According to the ...