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Rare Diseases

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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Using Technology that Powers GPS Cancer, Scientists have Developed Fresh Insights into the Driving Mechanisms of a Rare Skin Cancer

CULVER CITY, Calif.–(BUSINESS WIRE)–New, peer-reviewed research utilizing NantOmics technology, which powers NantHealth’s (NASDAQ: NH) GPS Cancer ® is giving scientists fresh insights into the driving mechanisms behind a form of rare skin cancer as well as therapies to treat patients. Scientists from NantOmics as well as the University of California …

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FDA Approves Diacomit for Treatment of Seizures Associated with Dravet Syndrome

GENTILLY, France, Aug. 23, 2018 /PRNewswire/ — BIOCODEX SAS is pleased to announce that on August 20th, 2018, the Food and Drug Administration (FDA) has approved DIACOMIT (stiripentol) for the treatment of seizures associated with Dravet syndrome (DS) in patients 2 years of age and older taking clobazam. DIACOMIT is …

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FDA Grants Rare Pediatric Disease Designation to Krystal’s KB105 for Patients with TGM-1-Deficient Autosomal Recessive Congenital Ichthyosis

PITTSBURGH, Aug. 23, 2018 (GLOBE NEWSWIRE) — Krystal Biotech, Inc. (Nasdaq: KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation (“RPDD”) to the company’s gene …

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