GUILDFORD, England, May 15, 2023 (GLOBE NEWSWIRE) — SiSaf Ltd, an RNA delivery and therapeutics company, announces that SIS-101-ADO, its siRNA therapeutic for patients with Autosomal Dominant Osteopetrosis Type 2 (ADO2), has been granted Orphan Drug Designation by the U.S. FDA. In addition, due to the serious manifestations of this...
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PRINCETON, N.J. — Soligenix, Inc. (Nasdaq: SNGX), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted orphan drug designation...
PRINCETON, NJ – Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the United States (U.S.) Food and Drug Administration...
THOUSAND OAKS, Calif. – Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for sotorasib for the treatment of patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), following at least one prior systemic therapy. The FDA grants Priority...
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...
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...