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Protalix and Chiesi to Apply for FDA Accelerated Approval of Pegunigalsidase Alfa for the Treatment of Fabry Disease

CARMIEL, Israel, June 06, 2019 (GLOBE NEWSWIRE) — Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), or Protalix, and Chiesi Farmaceutici S.p.A, an international research-focused healthcare Group, or Chiesi, today announced that, following a series of meetings and correspondence with the U.S. Food and Drug Administration (FDA), they plan to file …

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Takeda’s Ninlaro Fails to Meet Primary Endpoint in Phase 3 Trial in Patients with AL Amyloidosis

CAMBRIDGE, Mass. & OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) today announced that the Phase 3 TOURMALINE-AL1 clinical trial in patients with relapsed or refractory systemic light-chain (AL) amyloidosis did not meet the first of two primary endpoints. Treatment with NINLAROTM (ixazomib) in combination with dexamethasone did not demonstrate …

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Alnylam Completes Rolling Submission of Givosiran NDA to the FDA for Treatment of Acute Hepatic Porphyria

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment …

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FDA Grants Priority Review to Enzyvant’s RVT-802, an Investigational Tissue-Based Regenerative Therapy for Pediatric Congenital Athymia

CAMBRIDGE, Mass. & BASEL, Switzerland–(BUSINESS WIRE)–Enzyvant, a biopharmaceutical company focused on developing and commercializing transformative therapies for patients with rare, often fatal conditions, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its Biologics License Application (BLA) for RVT-802, a novel investigational tissue-based regenerative therapy …

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FDA Grants Fast Track Designation to SpringWorks’ Investigational Therapy for Treatment of a Severe Form of Neurofibromatosis Type 1

STAMFORD, Conn.–(BUSINESS WIRE)–SpringWorks Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PD-0325901, an investigational, oral, small molecule inhibitor of MEK1 and MEK2, for …

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Vertex Selects Triple Combination Regimen of VX-445, Tezacaftor and Ivacaftor to Submit for Global Regulatory Approvals in Cystic Fibrosis

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has selected the triple combination of the next-generation corrector VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential global regulatory approvals for people ages 12 and older with cystic fibrosis (CF). Final data announced today from a 24-week Phase 3 study …

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Fibrocell Receives FDA Regenerative Medicine Advanced Therapy Designation for FCX-007 Gene Therapy for Treatment of RDEB

EXTON, Pa., May 29, 2019 (GLOBE NEWSWIRE) — Fibrocell Science, Inc. (Nasdaq: FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to FCX-007, …

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FDA Grants Fast Track Designation to Imara’s IMR-687 for the Treatment of Sickle Cell Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Imara, Inc., a clinical-stage biopharmaceutical company focused on sickle cell disease and other hemoglobinopathies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to IMR-687, the company’s lead product candidate for the treatment of sickle cell disease. IMR-687 is currently being evaluated in a …

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FDA Grants Orphan Drug Designation to Applied Therapeutics AT-007 for Treatment of Galactosemia

NEW YORK, May 28, 2019 (GLOBE NEWSWIRE) — Applied Therapeutics Inc. (Nasdaq:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to …

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FDA Approves AveXis’ Zolgensma, the First and Only Gene Therapy for Pediatric Patients with Spinal Muscular Atrophy

Basel, May 24, 2019 – AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovac-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed …

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