Rare Diseases

Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in Mucopolysaccharidosis Type IIIA

PARIS–(BUSINESS WIRE)–Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). LYS-SAF302, …

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FDA Authorizes Marketing of the First Genetic Test to Aid in the Diagnosis of Fragile X Syndrome

The U.S. Food and Drug Administration today authorized marketing of the first test to detect a genetic condition known as Fragile X Syndrome (FXS), the most common known cause of inherited developmental delay and intellectual disability. The test is intended as an aid in diagnosing FXS and is to be …

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FORMA Therapeutics Announces FT-4202 Granted Fast Track Designation and Rare Pediatric Disease Designation for Treatment of Sickle Cell Disease

WATERTOWN, Mass.–(BUSINESS WIRE)–FORMA Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Rare Pediatric Disease designation for its lead clinical asset, FT-4202, in development as a disease-modifying treatment for sickle cell …

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Results from Phase 2 Study of Galderma’s Investigational Therapy Nemolizumab in Patients with Moderate to Severe Prurigo Nodularis

LAUSANNE, Switzerland–(BUSINESS WIRE)–Galderma today announced that The New England Journal of Medicine (NEJM) has published full results from the Phase 2 study of its investigational therapy nemolizumab in adult patients suffering from moderate-to-severe prurigo nodularis (PN). PN is a rare, potentially debilitating, chronic skin condition with thick skin nodules covering …

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Horizon Announces FDA Approval of New Dosage Form of Procysbi Delayed-Release Oral Granules for Nephropathic Cystinosis

DUBLIN–(BUSINESS WIRE)–Horizon Therapeutics plc (Nasdaq: HZNP) announced today that the U.S. Food and Drug Administration (FDA) has approved PROCYSBI® (cysteamine bitartrate) delayed-release oral granules in packets for adults and children one year of age and older living with nephropathic cystinosis. This new dosage form provides another option for people living …

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Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 With Type 2 or 3 Spinal Muscular Atrophy

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed …

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PTC Therapeutics Announces Publication of Real-World Data Shows Translarna Significantly Preserves Ability to Walk for Longer in Children with DMD

SOUTH PLAINFIELD, N.J., Feb. 3, 2020 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Pulmonary function was …

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Ipsen Pauses Palovarotene Program After Interim Analysis Finds Phase 3 Trial Unlikely to Meet its Primary Endpoint

Ipsen (Euronext: IPN; ADR: IPSEY) today announced the decision to pause dosing patients in the global Phase III (PVO-1A-301) study designed to evaluate the efficacy and safety of palovarotene in patients with fibrodysplasia ossificans progressiva (FOP), as well as the ongoing Phase II (PVO-1A-202/204) extension studies. In both the Phase …

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Genentech’s Risdiplam Meets Primary Endpoint in Pivotal Trial in Infants with Type 1 Spinal Muscular Atrophy

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the …

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FDA Approves Horizon’s Tepezza for Treatment of Thyroid Eye Disease

DUBLIN–(BUSINESS WIRE)–Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the U.S. Food and Drug Administration (FDA) has approved TEPEZZATM (teprotumumab-trbw) for the treatment of Thyroid Eye Disease (TED). TEPEZZA is the first and only FDA-approved medicine for the treatment of TED, a serious, progressive and vision-threatening rare autoimmune disease that …

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