BOSTON, Mass. — NeuroNOS, a biopharmaceutical company focused on developing treatments for neurological disorders and neuro-oncology, and a subsidiary of Beyond Air (NASDAQ: XAIR), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead investigational therapy, BA-101, for the treatment of...
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PARAMUS, N.J. — NS Pharma, Inc. announced today that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-051/NCNP-04 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne) in patients amenable to exon 51 skipping. The FDA issues Orphan Drug Designations to support...
PARAMUS, N.J. — NS Pharma, Inc. (NS Pharma) a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA)....
CAMBRIDGE, Mass. — Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for SH-110, a palatable oral suspension to treat Glioma – a rare brain cancer – by providing a liquid form of treatment for patients...
MARLBOROUGH, Mass. — Sumitomo Pharma America, Inc. (SMPA) today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to its investigational therapy DSP-3077 for the treatment of retinitis pigmentosa (RP). DSP-3077 is an investigative regenerative cell therapy that leverages allogeneic retinal sheets derived from induced pluripotent...
Ymmunobio, a preclinical stage biotech company specializing in the development of innovative treatments for cancer patients, announced today that it has been granted Orphan Drug Designation (ODD) by the US Food & Drug Administration (FDA) for YB-200, a CEACAM1/5 antibody it is studying for the treatment of hepatocellular carcinoma
Calgary, Alberta and Amsterdam, the Netherlands — Zenith Epigenetics Ltd. (“Zenith” or the “Company”) is pleased to announce that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug designation to ZEN-3694 for treatment of NUT carcinoma. NUT carcinoma is a rare, aggressive form of cancer with no currently...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S....
CAMBRIDGE, Mass. — Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is...
STOCKHOLM – Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”) today announced that the U.S. Food and Drug Administration (FDA) has accepted the submission and granted Priority Review for the New Drug Application (NDA) for Nefecon, a down regulator of IgA1 for the treatment of IgA nephropathy (IgAN.) The FDA...