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FDA Approves Gattex for Children One Year of Age and Older with Short Bowel Syndrome

LEXINGTON, Mass., May 17, 2019 /PRNewswire/ — Takeda Pharmaceuticals, U.S.A., Inc. (“Takeda”), announced today that the U.S. Food and Drug Administration (FDA) approved extending the indication of GATTEX® (teduglutide) for injection to pediatric patients 1 year of age and older with Short Bowel Syndrome (SBS) who need additional nutrition or …

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FDA Approves Broadened Indication for Xeomin as First-Line Treatment for Blepharospasm in Adult Patients

RALEIGH, N.C.–(BUSINESS WIRE)–Merz Americas announced today that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for XEOMIN® (incobotulinumtoxinA), broadening its indication to be a first-line treatment of blepharospasm (involuntary blinking) in adult patients. “Merz is proud to offer a first-line treatment option for blepharospasm, …

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Pfizer Acquires Clinical-Stage Biotech Therachon for up to $810 Million, Expanding Rare Disease Pipeline

NEW YORK & BASEL, Switzerland–(BUSINESS WIRE)–Pfizer (NYSE: PFE) today announced that it has entered into a definitive agreement to acquire all the shares of Therachon Holding AG, a privately-held clinical-stage biotechnology company focused on rare diseases, with assets in development for the treatment of achondroplasia and short bowel syndrome (SBS). Under …

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FDA Approves First Treatment for Children with Lambert-Eaton Myasthenic Syndrome, a Rare Autoimmune Disorder

The U.S. Food and Drug Administration today approved Ruzurgi (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. This is the first FDA approval of a treatment specifically for pediatric patients with LEMS. The only other treatment approved for …

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FDA Approves Vyndaqel and Vyndamax for Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™ (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral …

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FDA Grants Zynerba’s Zygel Fast Track Designation for the Treatment of Behavioral Symptoms Associated with Fragile X Syndrome

DEVON, Pa., May 06, 2019 (GLOBE NEWSWIRE) — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s lead development candidate Zygel™ (ZYN002 CBD …

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Alexion Announces Positive Phase 3 Data for Soliris in Patients with Neuromyelitis Optica Spectrum Disorder Published in NEJM

BOSTON & PHILADELPHIA–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the New England Journal of Medicine (NEJM) published positive data from the Phase 3 PREVENT study of SOLIRIS® (eculizumab), a first-in-class complement inhibitor, in adult patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare and devastating, autoimmune, inflammatory disorder …

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Post-Hoc Analyses of Radicava Studies Show Phase 3 Study Design Minimized ALS Heterogeneity to Improve Trial Efficiency

JERSEY CITY, N.J., May 2, 2019 /PRNewswire/ — Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced results from a post-hoc analysis that showed how the RADICAVA® (edaravone) Phase 3 study design minimized heterogeneity of the study population to successfully demonstrate a treatment effect in amyotrophic lateral sclerosis (ALS) in 24 …

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FDA Approves Kalydeco as First and Only CFTR Modulator to Treat Eligible Infants with Cystic Fibrosis as Early as 6 Months

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

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FDA Approves Convenient Single-Vial Dosing for CSL Behring’s Zemaira for Patients with Alpha 1 Antitrypsin Deficiency

KING OF PRUSSIA, Pa., April 25, 2019 /PRNewswire/ — Global biotherapeutics leader CSL Behring today announced that the US Food and Drug Administration (FDA) has approved 4- and 5-gram vial sizes for ZEMAIRA® [Alpha1-Proteinase Inhibitor (Human)], its therapy for treating Alpha 1 Antitrypsin Deficiency (Alpha 1).  This approval is significant …

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