Home / Rare Diseases (page 2)

Rare Diseases

FDA Approves ADMA Biologics’ Asceniv, a Novel Intravenous Immune Globulin

RAMSEY, N.J. and BOCA RATON, Fla., April 01, 2019 (GLOBE NEWSWIRE) — ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), a vertically integrated commercial biopharmaceutical and specialty immunoglobulin company that manufactures, markets and develops plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious diseases, …

Read More »

Sarepta Therapeutics Announces Positive Expression Results from the Casimersen Arm of the Phase 3 ESSENCE Study in DMD Patients

CAMBRIDGE, Mass., March 28, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced results from its interim analysis of muscle biopsy endpoints comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, …

Read More »

Proteostasis Announces Broad New Dataset from Proprietary Combination and Add-On CFTR Modulator Studies in Cystic Fibrosis

BOSTON, March 25, 2019 /PRNewswire/ — Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced results from Phase 1 clinical studies of its proprietary cystic …

Read More »

Enzyvant’s Investigational Farber Disease Enzyme Replacement Therapy Receives FDA Fast Track and Rare Pediatric Disease Designations

CAMBRIDGE, Mass. and BASEL, Switzerland, March 21, 2019 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing and commercializing transformative therapies for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease and Fast Track designations for RVT-801, an investigational enzyme …

Read More »

Alexion and Affibody Announce Partnership to Co-Develop Anti-FcRn Affibody Molecule

BOSTON & SOLNA, Sweden–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Affibody AB today announced a partnership to co-develop ABY-039 for rare Immunoglobulin G (IgG)-mediated autoimmune diseases. Currently in Phase 1 development, ABY-039 is a bivalent antibody-mimetic that targets the neonatal Fc receptor (FcRn). ABY-039 has been specifically designed to combine Affibody’s protein …

Read More »

Soleno Announces Positive Outcome from Planned Data Safety Monitoring Board Review of Phase 3 Trial of DCCR in Prader-Willi Syndrome

REDWOOD CITY, Calif.–(BUSINESS WIRE)–Soleno Therapeutics, Inc. (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the Data Safety Monitoring Board (DSMB) has recommended the continuation of the Company’s Phase III trial in Prader-Willi Syndrome (PWS) patients, DESTINY PWS, without any changes. …

Read More »

Alnylam Reports Positive Topline Results from Phase 3 Study of Givosiran in Acute Hepatic Porphyria

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today that the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), met its primary efficacy endpoint and the majority of secondary …

Read More »

Two Phase 3 Studies of the Triple Combination of VX-445, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function in Cystic Fibrosis Patients

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that treatment with the triple combination of the next-generation corrector VX-445, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF). …

Read More »

Celgene’s Fedratinib Granted Priority Review by the FDA for Treatment of Myelofibrosis

SUMMIT, N.J.–(BUSINESS WIRE)–Celgene Corporation (NASDAQ:CELG) today announced the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for fedratinib and granted a Priority Review. Fedratinib is a highly selective JAK2 inhibitor intended for the treatment of patients with myelofibrosis, a serious bone marrow disorder that …

Read More »

Acceleron Receives FDA Orphan Drug Designation for ACE-083 in Charcot-Marie-Tooth Disease

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 that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to ACE-083, the Company’s locally-acting “Myostatin+” muscle agent, for …

Read More »