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Alexion and Stealth Announce Agreement for Option to Co-Develop and Commercialize Late-Stage Therapy for Mitochondrial Diseases

BOSTON–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Stealth BioTherapeutics Corp (NASDAQ:MITO) today announced an agreement for an option to co-develop and commercialize elamipretide for mitochondrial diseases. Currently being evaluated in a Phase 3 study in people with primary mitochondrial myopathy (PMM) – a genetic mitochondrial disease – elamipretide is a novel, potential first-in-class therapy …

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ProQR Announces Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients

LEIDEN, Netherlands and CAMBRIDGE, Mass., Oct. 10, 2019 (GLOBE NEWSWIRE) — ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced positive top-line results from the PQ-110-001 study, a Phase 1/2 dose range finding, first-in-human trial …

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FDA Approves Clinuvel’s Scenesse, the First Treatment for a Rare Genetic Metabolic Disorder that Causes Light Intolerance

MELBOURNE, Australia, Oct. 08, 2019 (GLOBE NEWSWIRE) — The US Food and Drug Administration (FDA) has approved a new drug as the first ever treatment for a rare genetic metabolic disorder which causes absolute intolerance to light. SCENESSE® (afamelanotide 16mg), the first drug developed by Australian biopharmaceutical company CLINUVEL, has …

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Savara Announces FDA Response From Type C Meeting on Molgradex for aPAP Development Program

AUSTIN, Texas–(BUSINESS WIRE)–Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, today announced the response from a Type C meeting with the U.S. Food and Drug Administration (FDA) regarding the Molgradex development program for autoimmune pulmonary alveolar proteinosis (aPAP). Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). …

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Landmark Study of Infants with Spinal Muscular Atrophy Treated Pre-Symptomatically with Spinraza Published in Neuromuscular Disorders

CAMBRIDGE, Mass., Oct. 02, 2019 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment …

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NewLink Genetics and Lumos Pharma Enter into Merger Agreement to Form Company Focused on Developing Rare Disease Drugs

AUSTIN, Texas and AMES, Iowa, Sept. 30, 2019 (GLOBE NEWSWIRE) — NewLink Genetics Corporation (NASDAQ:NLNK) and Lumos Pharma, Inc., a private clinical stage biopharmaceutical company focused on development and commercialization of therapeutics for rare diseases, today announced that the companies have entered into a definitive merger agreement. Under the terms …

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Annexon Biosciences Reports Top-line Phase 1b Results for Novel C1q Inhibitor ANX005 in Guillain-Barré Syndrome

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Annexon Biosciences, a clinical-stage biopharmaceutical company developing a pipeline of novel therapies for patients with classical complement-mediated disorders of the body, eye and brain, today announced encouraging results from a Phase 1b dose-ranging clinical trial to evaluate its anti-C1q antibody, ANX005. The trial was conducted in …

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Ultragenyx and Kyowa Kirin Announce FDA Approves Label Update for Crysvita for the Treatment of X-Linked Hypophosphatemia

NOVATO, Calif., and TOKYO, Sept. 30, 2019 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) has approved a label …

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FDA Approves Genentech’s Rituxan in Children With Two Rare Blood Vessel Disorders

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Rituxan® (rituximab), in combination with glucocorticoids, for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in pediatric patients 2 years …

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Zogenix Resubmits its New Drug Application for Fintepla to the FDA for the Treatment of Dravet Syndrome

EMERYVILLE, Calif., Sept. 26, 2019 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that it has resubmitted its New Drug Application (NDA) for FINTEPLA® (ZX008, fenfluramine) for the treatment of seizures associated with Dravet syndrome to the U.S. Food and Drug …

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