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Abeona Therapeutics Receives FDA Fast Track Designation for ABO-202 AAV9 Gene Therapy in CLN1 Disease

NEW YORK and CLEVELAND, June 18, 2019 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its ABO-202 program. ABO-202, the Company’s novel one-time AAV9 gene therapy …

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Voyager Therapeutics Announces Restructured Gene Therapy Relationship with Sanofi Genzyme and Portfolio Update

CAMBRIDGE, Mass., June 17, 2019 (GLOBE NEWSWIRE) — Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced a restructuring of its gene therapy relationship with Sanofi Genzyme. Under the terms of the agreement, Voyager gains worldwide rights to …

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Updated Efficacy Data from GBT’s Phase 3 Study Show Statistically Significant and Sustained Improvements in Hemoglobin with Voxelotor

AMSTERDAM, The Netherlands, June 14, 2019 (GLOBE NEWSWIRE) — Global Blood Therapeutics, Inc. (GBT) (Nasdaq: GBT) today announced new results from its Phase 3 HOPE Study of voxelotor in patients ages 12 and older with sickle cell disease (SCD). The findings from 274 adolescents and adults treated with voxelotor showed the HOPE Study met …

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Phase 3 Study Showed that Genentech’s Rituxan is Superior to Standard of Care in Achieving Sustained Remission in Pemphigus Vulgaris

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced positive top line results from the Roche-sponsored Phase III PEMPHIX study evaluating the efficacy and safety of Rituxan® (rituximab) compared to mycophenolate mofetil (MMF) in adults with moderate to severe pemphigus vulgaris (PV). …

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Catalyst Pharmaceuticals Files a Federal Lawsuit Against the FDA Over Approval of Firdapse Competitor in LEMS

CORAL GABLES, Fla., June 12, 2019 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq:CPRX), today announced it has filed a suit against the U.S. Food and Drug Administration (FDA) and several related parties challenging the recent approval of a new drug application and related drug labeling for Jacobus Pharmaceutical Company’s …

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Genentech’s Rituxan Receives Priority Review from the FDA for Treatment of Children with Two Rare Blood Vessel Disorders

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for the use of Rituxan® (rituximab), in combination with glucocorticoids (GCC), for …

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FDA Grants Orphan Drug and Rare Pediatric Disease Designation for Denali’s DNL310 for Treatment of Hunter Syndrome

SOUTH SAN FRANCISCO, Calif., June 11, 2019 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases, today announced that the FDA granted Orphan Drug Designation (“ODD”) and Rare Pediatric Disease Designation for Denali’s DNL310 program, which Denali is …

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FDA Expands Approval of PTC Therapeutics’ Emflaza to Include Duchenne Muscular Dystrophy Patients 2-5 Years of Age

SOUTH PLAINFIELD, N.J., June 7, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) approved the company’s supplemental New Drug Application (sNDA) for Emflaza® (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy who are between 2- and 5-years-old. Duchenne is a rare childhood …

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Vertex Expands Collaboration with CRISPR Therapeutics and Acquires Exonics Therapeutics

BOSTON & WATERTOWN, Mass. & ZUG, Switzerland–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that the company is enhancing its gene editing capabilities to develop novel therapies for Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1) by expanding its collaboration with CRISPR Therapeutics and acquiring Exonics Therapeutics. Vertex …

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La Jolla Pharmaceutical Announces Positive Interim Results from Phase 2 Study of LJPC-401 in Patients with Hereditary Hemochromatosis

SAN DIEGO, June 06, 2019 (GLOBE NEWSWIRE) — La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced positive results from the pre-specified interim analysis of its Phase 2 study of LJPC-401 …

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