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Rare Diseases

Teva’s Investigational Tardive Dyskinesia Drug Granted FDA Breakthrough Therapy Designation

JERUSALEM–(BUSINESS WIRE)–Teva Pharmaceutical Industries Ltd. (NYSE and TASE:TEVA) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to SD-809 (deutetrabenazine) for the treatment of patients with moderate to severe tardive dyskinesia, a hyperkinetic movement disorder affecting about 500,000 people in the United States. …

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Agilis Biotherapeutics’ Investigational Angelman Syndrome Drug Granted Orphan Drug Status

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AGIL-AS, the Company’s gene therapy product candidate being developed for …

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Shire to Acquire Dyax for up to $6.5 Billion

Shire plc (LSE: SHP, NASDAQ: SHPG) and Dyax Corp. (NASDAQ: DYAX) today announced that Shire will acquire Dyax for $37.30 in cash per Dyax share, for aggregate upfront consideration of approximately $5.9 billion. Dyax shareholders may receive additional value through a non-tradable contingent value right (CVR) that will pay $4.00 …

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Daiichi Sankyo’s Rare Cancer Drug Receives Breakthrough Therapy Designation from the FDA

Parsippany, NJ and Berkeley, CA – (October 30, 2015) – Daiichi Sankyo, Inc. and Plexxikon Inc., a member of the Daiichi Sankyo Group, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its investigational oral CSF-1R inhibitor pexidartinib (formerly PLX3397) for the treatment …

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Ultragenyx and Arcturus Enter Potential $1.57 Billion RNA Collaboration

NOVATO, Calif., Oct. 29, 2015 /PRNewswire/ — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Arcturus Therapeutics, Inc., a leading RNA medicines company, today announced that they have entered into a research collaboration and license agreement to discover …

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Alexion Wins FDA Approval of Strensiq and Priority Review Voucher

Today, the U.S. Food and Drug Administration approved Strensiq (asfotase alfa) as the first approved treatment for perinatal, infantile and juvenile-onset hypophosphatasia (HPP). HPP is a rare, genetic, progressive, metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to severe disability and life-threatening complications. …

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Precision Medicine Approach Helps Identify and Treat Toddler’s Rare Disease

Newswise — NEW YORK, NY (October 22, 2015) — A 20-month-old girl suffering from a rare neurodegenerative disease was diagnosed by exome sequencing and successfully treated. The case, which exemplifies the potential of precision medicine, involved scientists at Columbia University Medical Center (CUMC) and Duke University, and is described in …

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FDA Approves First Factor X Concentrate for Treatment of a Rare Hereditary Bleeding Disorder

The U.S. Food and Drug Administration today approved Coagadex, Coagulation Factor X (Human), for hereditary Factor X (10) deficiency. Until today’s orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency. In healthy individuals, the Factor X protein activates enzymes to help …

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PTC Announces Late-Stage Results for its Duchenne Muscular Dystrophy Candidate

SOUTH PLAINFIELD, N.J., Oct. 15, 2015 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 3, double-blind, placebo-controlled, 48-week ACT DMD trial of Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).  The trial results showed clinically …

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Organ Transplant Drug Linked to Increased Risk of Rare Lymphoma

A study led by Johns Hopkins researchers has linked the immunosuppressive drug mycophenolate mofetil (MMF) to an increased risk of central nervous system (CNS) lymphoma in solid organ transplant patients. But the same study also found that another class of immunosuppressive drugs, called calcineurin inhibitors (CNIs), given alone or in …

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