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Alnylam to Initiate Rolling Submission of NDA and Pursue Full Approval for Givosiran for Acute Hepatic Porphyria

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that in consultation with the U.S. Food and Drug Administration (FDA), the Company plans to pursue a full approval based on the complete results of the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting …

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New Data Shows PTC Therapeutics’ Translarna Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation

SOUTH PLAINFIELD, N.J., Oct. 6, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense …

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FDA Grants Orphan Drug Designation to MediciNova’s Investigational Glioblastoma Candidate

LA JOLLA, Calif., Oct. 04, 2018 (GLOBE NEWSWIRE) — MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number:4875), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to MN-166 (ibudilast) as …

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FDA Grants Fast Track Designation to Protagonist’s Hepcidin Mimetic PTG-300

NEWARK, Calif., Sept. 27, 2018 /PRNewswire/ — Protagonist Therapeutics, Inc. (Nasdaq:PTGX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to therapeutic candidate PTG-300 for the treatment of chronic anemia due to ineffective erythropoiesis in patients with beta-thalassemia. PTG-300 is an injectable hepcidin mimetic that has been granted Orphan Drug …

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Alexion to Acquire Syntimmune for up to $1.2 Billion

BOSTON–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Syntimmune today announced that they have entered into a definitive agreement for Alexion to acquire Syntimmune, a clinical-stage biotechnology company developing antibody therapeutics targeting the neonatal Fc receptor (FcRn). SYNT001 – a humanized monoclonal antibody that inhibits the interaction of FcRn with Immunoglobulin G (IgG) …

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Alexion’s Soliris Demonstrates Positive Results in Phase 3 Study in Patients with Neuromyelitis Optica Spectrum Disorder

BOSTON–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced positive topline results from the Phase 3 PREVENT study of Soliris® (eculizumab) in patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses. Each relapse results in stepwise …

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Catabasis to Present New Edasalonexent Clinical Results and Phase 3 Trial Design in Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Company will share three presentations, including in the late breaking session, at the 23rd International Congress of the World Muscle Society, being held October 2 – 6, 2018, at the Intercontinental Hotel in Mendoza, Argentina. Presentations include: Late …

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Pfizer Presents New Analyses from the Tafamidis Phase 3 Transthyretin Amyloid Cardiomyopathy Study

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) announced today that additional sensitivity and post-hoc analyses from the Tafamidis Phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study provide further detail on the effect of tafamidis across wild-type, hereditary, and New York Heart Association (NYHA) class sub-groups of patients with transthyretin amyloid cardiomyopathy (ATTR-CM).1 Tafamidis is …

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FDA Grants Fast Track Designation to ArQule’s Miransertib for the Treatment of PIK3CA-Related Overgrowth Spectrum

BURLINGTON, Mass.–(BUSINESS WIRE)–ArQule, Inc. (Nasdaq: ARQL) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to miransertib (ARQ 092) for the treatment of PIK3CA-Related Overgrowth Spectrum (PROS), a group of related, ultra-rare genetic disorders characterized by excessive tissue growth in various parts of the …

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After Phase 2 Failure, Pfizer Terminates Domagrozumab Program for Treatment of Duchenne Muscular Dystrophy

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004). The Phase 2 study (B5161002), did not meet its …

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