LEHI, Utah — Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company pioneering therapies that target the root causes of inflammation-driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational medicine ofirnoflast (HT-6184) for the treatment of Myelodysplastic Syndromes (MDS) — a group...
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CHARLESTON, S.C. — Leukogene Therapeutics Inc. (LTI), a biopharmaceutical company developing next-generation immunotherapies for hematologic and other malignancies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the Company’s lead product candidate, M2T-CD33 (LTI-214), for the treatment of Acute Myeloid Leukemia (AML). This...
SHANGHAI, China — Mabwell (688062.SH), an innovative biopharmaceutical company with entire industry chain, announced that FDA has granted Orphan Drug Designation (ODD) to 9MW3011 (R&D code in the US: MWTX-003/DISC-3405) for the treatment of patients with polycythemia vera (PV). FDA Orphan Drug Designation is granted to investigational therapies addressing rare...
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...
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...
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....