Tokyo, Japan – The FDA on Tuesday approved the use of Takeda’s HyQvia (immune globulin infusion 10% with recombinant human hyaluronidase) as a treatment for chronic inflammatory demyelinating polyneuropathy. With the label expansion, HyQvia can now be given as a maintenance therapy to prevent the relapse of neuromuscular disability and...
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DAVIS, Calif. — Infinant Health, a privately-held company focused on changing the trajectory of human health – one baby at a time – announced today that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to its investigational drug...
ROCKVILLE, Md. — NeoImmuneTech, Inc. (NIT), a T cell-focused therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has granted NT-I7 (efineptakin alfa) (rhIL-7-hyFc) Orphan Drug Designation (ODD) for the treatment of pancreatic cancer. Pancreatic cancer is an aggressive tumor-type associated with extremely poor prognosis. It is the...
WALTHAM, Mass. — Affinia Therapeutics (“Affinia”), an innovative clinical-stage gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for AFTX-201 for the treatment of...
Sarasota, Florida — BIOM Pharmaceutical Corporation is thrilled to announce that Bi104, its innovative drug product, has been granted orphan-drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome (AS). Angelman Syndrome (AS) is a rare neurodevelopmental disorder characterized by developmental delay, intellectual disability,...
BOSTON, Mass. — Dewpoint Therapeutics, the leader in condensate biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to DPTX3186, its first-in-class condensate modulator for the treatment of gastric cancer. The designation follows the recent opening of Dewpoint’s Investigational New Drug (IND)...
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...
Wilmington, Delaware – The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to IKT-001, an investigational formulation of imatinib developed by Inhibikase Therapeutics for the treatment of pulmonary arterial hypertension (PAH). The designation is intended to encourage the development of therapies for rare diseases affecting fewer than...
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...