How far would you go to save your child’s life? Haoyang was born in Kunming in southwestern China two years ago with a very rare disease: Menkes Syndrome. The disease is a genetic disorder that impacts how copper – which is crucial for brain and nervous system development – is...
Latest News
Nashville, Tennessee – Among adult patients with severe sickle cell disease (SCD), favorable outcomes were reported with use of a modified transplantation procedure, according to results from a multicenter study presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition in December 2023. Although the use of...
EMERYVILLE, Calif. — XOMA Corporation the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration’s (FDA) acceptance of Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol, an orally-delivered, first-in-class therapy for Niemann-Pick disease type C (NPC), XOMA will make a $1 million milestone payment to...
Basel, Switzerland – Roche (SIX: RO, ROP; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Gazyva®/Gazyvaro® (obinutuzumab) for the treatment of systemic lupus erythematosus (SLE). The filing acceptance is based on positive results from the phase...
AUSTIN, Texas — IntraBio Inc., a global biopharmaceutical company focused on developing and commercializing therapies for neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its supplemental New Drug Application (sNDA) for AQNEURSA® (levacetylleucine) for the treatment of Ataxia-Telangiectasia (A-T) in adults and...
Durham, N.C. – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AB-1009, an adeno-associated virus (AAV) gene therapy being developed for...
Stockholm, Sweden – Sobi® (STO: SOBI), a global biopharmaceutical company dedicated to delivering innovative treatments for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) seeking approval for Nanoecapsulated Sirolimus plus Pegadricase (NASP), formerly SEL-212, for the treatment...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the company’s PALYNZIQ® (pegvaliase-pqpz) supplemental Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU). The FDA has set a Prescription Drug User...
PRINCETON, N.J. — Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for iberdomide combined with standard treatment (daratumumab + dexamethasone – IberDd) in patients with relapsed or refractory multiple myeloma (RRMM). Iberdomide is part of an investigational, new...
WALTHAM, Mass. and BOULDER, Colo. — Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has agreed to accept its New Drug Application (NDA) for bezuclastinib in combination with sunitinib for patients with...