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Viela Bio’s Inebilizumab Receives Breakthrough Therapy Designation from the FDA for Treatment of Neuromyelitis Optica Spectrum Disorder

GAITHERSBURG, Md.–(BUSINESS WIRE)–Viela Bio today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for the Company’s anti-CD19 monoclonal antibody inebilizumab, an investigational monotherapy for neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, life-threatening autoimmune disease affecting the central nervous system. “The Breakthrough …

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St. Jude Gene Therapy Cures Babies with Bubble Boy Disease

MEMPHIS, Tenn., April 17, 2019 /PRNewswire/ — Gene therapy developed at St. Jude Children’s Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time. The results appear …

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Ultragenyx’s UX007 Granted FDA Fast Track Designation and Rare Pediatric Disease Designation for Treatment of Long-Chain Fatty Acid Oxidation Disorders

NOVATO, Calif., April 16, 2019 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Rare Pediatric Disease designation …

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FibroGen Receives Orphan Drug Designation from the FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy

SAN FRANCISCO, April 15, 2019 (GLOBE NEWSWIRE) — FibroGen, Inc. (NASDAQ: FGEN), a leading biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients …

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Fibrocell and Castle Creek Pharmaceuticals Collaborate for Development and Commercialization of FCX-007 Gene Therapy

EXTON, Pa., April 15, 2019 (GLOBE NEWSWIRE) — Fibrocell Science, Inc. (Nasdaq: FCSC), a cell and gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today announced the completion of a collaboration agreement with Castle Creek Pharmaceuticals to develop and commercialize Fibrocell’s lead gene …

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Zogenix Receives Refusal to File Letter from the FDA for its Fintepla NDA for Treatment of Seizures Associated with Dravet Syndrome

EMERYVILLE, Calif., April 08, 2019 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for FINTEPLA® (ZX008, fenfluramine hydrochloride) for …

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Celgene and Acceleron Submit Luspatercept BLA to the FDA for Myelodysplastic Syndromes and Beta-Thalassemia Indications

SUMMIT, N.J. & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that Celgene has submitted a Biologics License Application (BLA) for luspatercept, an erythroid maturation agent, for the treatment of adult patients with very low to intermediate risk myelodysplastic syndromes (MDS)-associated anemia who have …

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Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington’s Disease

CHICAGO, April 5, 2019 /PRNewswire/ — Scientist from Dystrogen Therapeutics Corp. will present data supporting a potential cutting-edge therapy for neurodegenerative diseases caused by trinucleotide repeats, such as Huntington’s disease (HD) and spinocerebellar ataxias (SCAs). Early data confirmed that the therapy has the ability to selectively silence only the mutated …

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Acceleron Discontinues Development of its Phase 1 Neuromuscular Disorders Molecule ACE-2494

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced it is discontinuing development of ACE-2494, a systemic muscle agent the company had been studying in a Phase 1 healthy volunteer trial for …

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Abeona Therapeutics Receives FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)

NEW YORK and CLEVELAND, April 04, 2019 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy development, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ABO-101, the Company’s novel one-time gene therapy for Sanfilippo syndrome …

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