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Rare Diseases

FDA Grants Orphan Drug Designation to PhaseRx’s Rare Liver Disorder Drug

SEATTLE, Nov. 28, 2016 /PRNewswire/ — PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in children, today announced that its lead candidate, PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the U.S. …

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Agilis’ AADC Gene Therapy Candidate Receives Rare Pediatric Disease Designation from the FDA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative gene therapies for rare genetic diseases that affect the central nervous system (CNS), announced today that the Food and Drug Administration (FDA) has granted a Rare Pediatric Disease (RPD) designation for its gene therapy product candidate, AGIL-AADC for the …

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FDA Grants Orphan Drug Designation to Genexine’s Investigational Growth Hormone Deficiency Drug

SEONGNAM, South Korea–(BUSINESS WIRE)–Genexine (KOSDAQ: 095700), an innovative biotechnology company focused on immuno-oncology, metabolic and orphan diseases, announced today that US FDA Office of Orphan Products Development (OOPD) has granted GX-H9 an orphan drug designation for the treatment of growth hormone deficiency. GX-H9 is next-generation, long-acting recombinant human growth hormone …

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FDA Grants Orphan Drug Designation to Eloxx’s Enzyme Deficiency Drug

NESS ZIONA, Israel and WALTHAM, Massachusetts, November 7, 2016 /PRNewswire/ — Eloxx Pharmaceuticals, a biotechnology company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations, announced today that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) …

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WSU Researcher Develops Safer Gene Therapy

SPOKANE, Wash. – A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy. Grant Trobridge, an associate professor of pharmaceutical sciences, has altered the way a virus carries a beneficial gene to its target …

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Eisai Initiates Two Phase 3 Trials with Fycompa in Children with Epilepsy

WOODCLIFF LAKE, N.J., Oct. 28, 2016 /PRNewswire/ — Eisai Inc. announced today the initiation of two multi-center, global Phase 3 clinical trials assessing FYCOMPA® (perampanel) CIII in two different patient populations: patients age 2 years and above with inadequately controlled seizures associated with Lennox-Gastaut Syndrome (LGS) (Study 338), a rare …

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Solid Biosciences’ Gene Therapy Granted Orphan Drug Status for Duchenne Muscular Dystrophy

CAMBRIDGE, Mass. & LONDON–(BUSINESS WIRE)–Solid Biosciences and its subsidiary, Solid GT, announced today that the U.S. Food and Drug Administration and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD). Solid plans to initiate …

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FDA Accepts Resubmission of Teva’s Huntington Disease Drug

JERUSALEM–(BUSINESS WIRE)–Teva Pharmaceutical Industries Ltd. (NYSE and TASE:TEVA) today announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the New Drug Application (NDA) for SD-809 (deutetrabenazine) for the treatment of chorea associated with Huntington disease (HD). The FDA has assigned a Prescription Drug User Fee …

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FDA Awards 21 Grants to Stimulate Product Development for Rare Diseases

The U.S. Food and Drug Administration today announced that it has awarded 21 new clinical trial research grants totaling more than $23 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and …

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PTC Therapeutics Provides Regulatory Update on Translarna for Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., Oct. 17, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a regulatory update on Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). U.S. regulatory update PTC Therapeutics announced today that at the end of last week, the Office of Drug Evaluation …

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