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Wave Life Sciences’ WVE-210201 Granted Orphan Drug and Rare Pediatric Disease Designations from the FDA for Treatment of DMD

CAMBRIDGE, Mass., Aug. 16, 2018 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for …

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FDA Approves Kalydeco for Treatment of Cystic Fibrosis in Children Ages 12 to <24 Months with Certain Mutations in the CFTR Gene

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO® (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to <24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO …

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FDA Grants Orphan Drug Designation to Onspira’s Investigational Bronchiolitis Obliterans Therapy

WAYNE, Pa.–(BUSINESS WIRE)–Onspira Therapeutics, a privately-held clinical-stage biopharmaceutical company dedicated to developing therapies to treat rare, life threatening pulmonary diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its investigational product, OSP-101, for the treatment of Bronchiolitis Obliterans (BO). BO is a progressive …

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Cellectar’s CLR 131 Receives FDA Rare Pediatric Disease Designation for the Treatment of Ewing’s Sarcoma

MADISON, Wis., Aug. 13, 2018 (GLOBE NEWSWIRE) — Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, announces today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131, …

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FDA Approves Amicus’ Galafold for Treatment of Adults with Fabry Disease

CRANBURY, N.J., Aug. 10, 2018 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Galafold™ (migalastat) 123 mg capsules. Galafold is an oral, precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha …

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FDA Approves Alnylam’s RNAi Therapeutic Onpattro for Treatment of Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) approved ONPATTRO™ (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. ONPATTRO is the …

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FDA Grants Prometic’s Rare Pediatric Disease Designation for its Alström Syndrome Drug

LAVAL, QC, Aug. 7, 2018 /PRNewswire/ – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“Prometic”) today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to its small molecule drug candidate, PBI-4050, for the treatment of Alström syndrome (AS). In addition to …

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Allergan and Editas Medicine to Jointly Develop CRISPR Genome Editing Experimental Medicine EDIT-101

DUBLIN, Ireland and CAMBRIDGE, Mass., Aug. 06, 2018 (GLOBE NEWSWIRE) — Allergan plc (NYSE: AGN), a leading global pharmaceutical company, and Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, today announced that Allergan’s wholly-owned subsidiary, Allergan Pharmaceuticals International Limited (Allergan), has exercised its option to develop and commercialize …

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FDA Grants Orphan Drug Designation to Pliant’s Investigational Idiopathic Pulmonary Fibrosis Therapy

SOUTH SAN FRANCISCO, Calif., Aug. 6, 2018 /PRNewswire/ — Pliant Therapeutics, Inc., a biotechnology company focused on discovering, developing and commercializing treatments for fibrotic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the company’s anti-fibrotic lead compound, PLN-74809, for the treatment …

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Mallinckrodt Forms Collaborative Research Partnership with Washington University Focused on Rare Diseases

STAINES-UPON-THAMES, United Kingdom, Aug. 6, 2018 /PRNewswire/ — Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced it has formed a collaborative research partnership with Washington University School of Medicine in St. Louis, a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News …

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