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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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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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