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Rare Diseases

FDA Approves GSK’s Nucala for the Treatment of Eosinophilic Granulomatosis with Polyangiitis

GlaxoSmithKline plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Nucala (mepolizumab) as the first targeted treatment for eosinophilic granulomatosis with polyangiitis (EGPA), previously known as Churg-Strauss syndrome.  GSK submitted a supplemental Biologics License Application (sBLA) for mepolizumab, an interleukin-5 (IL-5) antagonist, in June …

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FDA Expands Orphan Drug Designation for Alnylam’s Patisiran to Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today the U.S. Food and Drug Administration (FDA) has granted a request to amend the orphan drug designation for patisiran to the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis). This is an expansion to patisiran’s prior designation which was for …

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MeiraGTx’s Leber’s Congenital Amaurosis Drug Receives Rare Pediatric Disease Designation from the FDA

LONDON and NEW YORK, Dec. 7, 2017 /PRNewswire/ — MeiraGTx, a London and New York-based gene therapy company, today announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted rare pediatric disease designation to the Company’s gene therapy product …

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Imbruvica in Combination with Rituximab Meets Primary Endpoint in Phase 3 Waldenström’s Macroglobulinemia Study

RARITAN, N.J., Dec. 5, 2017 /PRNewswire/ — Janssen Research & Development, LLC (Janssen) announced that the Phase 3 iNNOVATE (PCYC-1127) study evaluating IMBRUVICA® (ibrutinib) in combination with rituximab (RITUXAN®) in relapsed/refractory and treatment-naïve patients with Waldenström’s macroglobulinemia (WM) successfully met its primary endpoint of progression-free survival (PFS). An Independent Data …

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Study Evaluates Refractory Epilepsy Screening Tool for Lennox-Gastaut Syndrome

DEERFIELD, Ill.–(BUSINESS WIRE)–Lundbeck today presented data at the American Epilepsy Society (AES) Annual Meeting in Washington, D.C. that demonstrates that the Refractory Epilepsy Screening Tool for LGS (REST-LGS) may help healthcare professionals identify patients who may benefit from further clinical evaluation for Lennox-Gastaut syndrome (LGS). The REST-LGS was developed by …

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FDA Grants Breakthrough Therapy and Orphan Drug Designation to PellePharm’s Gorlin Syndrome Drug

MENLO PARK, Calif.–(BUSINESS WIRE)–PellePharm, a clinical-stage biopharmaceutical company committed to targeting rare genetic dermatological conditions at the source, today announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation and Orphan Drug Designation to topical patidegib for reduction of the life-long serious clinical morbidity and …

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FDA Approves Ultragenyx’s Mepsevii for Treatment of a Rare, Genetic Disease

NOVATO, Calif., Nov. 15, 2017 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved MEPSEVII™ (vestronidase alfa), the first medicine approved for the treatment of …

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CSL’s Hizentra Meets Primary Endpoint in Phase 3 Study in Patients with Chronic Inflammatory Demyelinating Polyneuropathy

KING OF PRUSSIA, Pa., Nov. 9, 2017 /PRNewswire/ — Global biotherapeutics leader CSL Behring announced that results were published in The Lancet Neurology from its PATH study (Polyneuropathy And Treatment with Hizentra®), a pivotal Phase III study evaluating the safety, efficacy and tolerability of Hizentra (Immune Globulin Subcutaneous [Human] 20% …

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FDA Approves Genentech’s Zelboraf for Treatment of Patients with a Rare Blood Cancer

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Zelboraf® (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD is a rare, serious blood disease characterized by the abnormal multiplication of …

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Alnylam’s Rare Disease Drug Demonstrates Positive Results in Ongoing Phase 1/2 Study

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive preliminary data from its ongoing Phase 1/2 study with lumasiran (formerly known as ALN-GO1), an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). Results were presented today at …

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