NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “Our...
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MELBOURNE, Australia — Neurizon Therapeutics Limited (ASX: NUZ & NUZOA) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to announce that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its lead investigational drug, NUZ‑001. This decision marks a...
CAMBRIDGE, Mass.— Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) notified Sarepta that it may lift its voluntary pause on shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory patients with Duchenne. Sarepta will resume shipping ELEVIDYS...
CLEVELAND, Ohio — Abeona Therapeutics Inc. announced a regulatory update for prademagene zamikeracel (pz-cel). The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to the Company’s Biologics License Application (BLA) for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB)....
Washington. DC – The FDA has denied approval of Theratechnologies’ F8 formulation of tesamorelin, which the Canadian biotech was proposing to reduce excess abdominal fat in patients with HIV and lipodystrophy, the company announced Wednesday. In its Complete Response Letter, the regulator pointed to issues regarding tesamorelin’s chemistry, manufacturing and controls,...
ROCKVILLE, Md. — The U.S. Food and Drug Administration today warned health care professionals about the risk of serious liver injury associated with the use of the anti-thyroid drug propylthiouracil (PTU) for the treatment of Graves’ disease. “After analyzing adverse event reports, the FDA has identified an increased risk of...
Washington DC – The US Food and Drug Administration (FDA) issued a warning against the purchase and use of blood glucose management product that contain glyburide and metformin according to laboratory tests. According to an FDA statement last week, Dr. Ergin’s SugarMD Advanced Glucose Support should be avoided for its...
Reata Pharmaceuticals used patient histories with trials data to win approval for new Friedreich’s ataxia drug
New York, NY – An iron-binding drug that is already approved for treatment of other diseases could provide a novel way to attack ovarian tumors, according to a new study led by Weill Cornell Medicine researchers. The preclinical study, which combined the analysis of human ovarian tumors and animal models...
Creating a strategy to discontinue eculizumab in patients with atypical hemolytic uremic syndrome (aHUS) is reasonable and safe and improves quality of life, according to research in Blood. The strategy should be based on complement genetics. Eculizumab has drastically improved outcomes for patients with aHUS. There’s no clearly defined optimal duration...