ISTURISA® is a cortisol synthesis inhibitor that blocks the enzyme 11β-hydroxylase to help normalize hypercortisolemia in Cushing’s syndrome, a rare endocrine condition that can have significant impact on patients and their families The expanded indication is supported by an extensive clinical development program Milan, Italy and Bridgewater, NJ, – Today...
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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...
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...
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...
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...