Rare Diseases

Forty Seven and Rocket Pharmaceuticals Announce Research Collaboration for Fanconi Anemia

MENLO PARK, Calif. and NEW YORK, March 11, 2020 (GLOBE NEWSWIRE) — Forty Seven, Inc. (Nasdaq: FTSV) and Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) announced today that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 …

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AVROBIO Receives Orphan Drug Designation from the FDA for AVR-RD-04 for Treatment of Cystinosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s investigational gene therapy, AVR-RD-04, for the treatment of …

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FDA Approves Novartis’ Isturisa for Treatment of Adults with Cushing’s Disease

The U.S. Food and Drug Administration today approved Isturisa (osilodrostat) oral tablets for adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. Cushing’s disease is a rare disease in which the adrenal glands make too much of the …

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TG Therapeutics’ Umbralisib Receives Orphan Drug Designation from the FDA for Treatment of Follicular Lymphoma

NEW YORK, March 05, 2020 (GLOBE NEWSWIRE) — TG Therapeutics, Inc. (NASDAQ: TGTX) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to umbralisib, the Company’s investigational dual inhibitor of PI3K-delta and CK1-epsilon, for the treatment of patients with follicular lymphoma (FL). Umbralisib is being evaluated across several …

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FDA Grants Rare Pediatric Disease Designation for Trofinetide for the Treatment of Rett Syndrome

SAN DIEGO & MELBOURNE, Australia–(BUSINESS WIRE)–ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) and Neuren Pharmaceuticals Limited (ASX: NEU) announced today that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder. Upon FDA approval of …

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FDA Grants Rare Pediatric Disease Designation to Stealth BioTherapeutics’ Elamipretide for Treatment of Barth Syndrome

BOSTON, March 3, 2020 /PRNewswire/ — Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for elamipretide for the …

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FDA Accepts and Grants Priority Review to Crysvita sBLA for Treatment of Tumor-Induced Osteomalacia

NOVATO, Calif. and TOKYO, Feb. 27, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. …

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FDA Delays PDUFA Target Action Date for Zogenix’s Fintepla for Treatment of Dravet Syndrome

EMERYVILLE, Calif., Feb. 27, 2020 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for FINTEPLA® for the treatment of seizures associated with …

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FDA Grants Fast Track Designation for Translate Bio’s MRT5005 for the Treatment of Cystic Fibrosis

LEXINGTON, Mass., Feb. 26, 2020 (GLOBE NEWSWIRE) — Translate Bio, Inc. (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast …

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Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in Mucopolysaccharidosis Type IIIA

PARIS–(BUSINESS WIRE)–Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). LYS-SAF302, …

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