Rare Diseases

Protalix and Chiesi Submit BLA to the FDA for Pegunigalsidase Alfa for the Treatment of Fabry Disease

CARMIEL, Israel, May 28, 2020 /PRNewswire/ —  Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, or the Company, together with its development and commercialization partner Chiesi …

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FDA Grants Rare Pediatric Disease Designation to Azitra’s ATR-12 for Treatment of Netherton Syndrome

BRANFORD, Conn.–(BUSINESS WIRE)–Azitra, Inc., a clinical-stage medical dermatology company addressing serious skin conditions by harnessing the microbiome, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to ATR-12 for the treatment of Netherton syndrome (NS), a chronic genetic skin disease that can be life …

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FDA Grants Priority Review to Alnylam’s Lumasiran for the Treatment of Primary Hyperoxaluria Type 1

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the …

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FDA Approves Takeda’s Alunbrig as a First-Line Treatment for Patients Diagnosed with Rare and Serious Form of Lung Cancer

CAMBRIDGE, Mass. & OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) approved ALUNBRIG (brigatinib) for adult patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test. This approval expands ALUNBRIG’s current indication to include the …

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Part A of Phase 3 Trial of Dupixent for Treatment of Eosinophilic Esophagitis Meets Co-Primary Endpoints

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced positive results from Part A of the pivotal Phase 3 trial evaluating Dupixent® (dupilumab) in patients 12 years and older with eosinophilic esophagitis (EoE). The trial met both of its co-primary endpoints, as well as all key secondary endpoints. Dupixent is the first …

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Passage Bio Receives Rare Pediatric Disease Designation from the FDA for PBGM01 for Patients with GM1 Gangliosidosis

PHILADELPHIA, May 21, 2020 (GLOBE NEWSWIRE) — Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to PBGM01 broadly for the treatment …

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FDA Grants Priority Review to Eiger’s NDA for Zokinvy for Treatment of Progeria and Progeroid Laminopathies

PALO ALTO, Calif., May 19, 2020 /PRNewswire/ — Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for accelerated review of Zokinvy™ (lonafarnib) for …

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Innovent Biologics and MD Anderson Partner to Develop anti-PD-1 Therapy Tyvyt in Rare Cancers

SUZHOU, China, and HOUSTON, May 18, 2020 /PRNewswire/ — Innovent Biologics, Inc. (“Innovent”) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, autoimmune, metabolic and other major diseases, and The University of Texas MD Anderson Cancer Center today announced a strategic …

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Ultragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia

NOVATO, Calif., May 15, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the …

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Seelos Therapeutics Receives Rare Pediatric Disease Designation for SLS-005 for Treatment of Sanfilippo Syndrome

NEW YORK, May 15, 2020 (GLOBE NEWSWIRE) — Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced it has been granted Rare Pediatric Disease Designation (RPDD) for SLS-005 in Sanfilippo syndrome from the U.S. …

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