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Ultragenyx and Kyowa Kirin Submit sBLA to the FDA for Crysvita for Tumor-Induced Osteomalacia

NOVATO, Calif. and TOKYO, Jan. 13, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. …

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FDA Grants Orphan Drug Designation to Dova’s Avatrombopag for the Treatment of Chemotherapy-Induced Thrombocytopenia

DURHAM, N.C., Jan. 13, 2020 /PRNewswire/ — Dova Pharmaceuticals, Inc., a wholly owned subsidiary of Swedish Orphan Biovitrum AB (publ) (Sobi™), has been granted Orphan Drug Designation1 (ODD) by the US Food and Drug Administration (FDA) for avatrombopag for the potential treatment of chemotherapy-induced thrombocytopenia (CIT). Enrollment remains ongoing for …

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Alnylam Initiates Rolling Submission of NDA to FDA for 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 Company has initiated a rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria …

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Ultragenyx Announces Positive Topline Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase Deficiency

NOVATO, Calif., Jan. 09, 2020 (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 topline positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an …

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Regeneron Announces Encouraging Garetosmab Phase 2 Results in Patients with Ultra-Rare Debilitating Bone Disease

TARRYTOWN, N.Y., Jan. 9, 2020 /PRNewswire/ —  Approximately 25% decrease in total bone lesions (both new and existing) driven by nearly 90% reduction in formation of new lesions in patients with fibrodysplasia ossificans progressiva (FOP) Regeneron plans to discuss regulatory submission with regulatory authorities; pediatric trial planning underway Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced results …

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FDA Grants Rare Pediatric Disease Designation for Aruvant’s ARU-1801 for the Treatment of Sickle Cell Disease

NEW YORK and BASEL, Switzerland, Jan. 8, 2020 /PRNewswire/ — Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to ARU-1801, Aruvant’s investigational therapy for …

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New Results Published that Further Support Masitinib’s Potential Mode of Action in Amyotrophic Lateral Sclerosis

AB Science SA (NYSE Euronext – FR0010557264 – AB) today announced the publication by an international team of researchers of a previously unknown mechanism linked to the progression of Amyotrophic Lateral Sclerosis (ALS) that further reinforces the rationale for masitinib’s potential neuroprotective effect in ALS. The publication, led by researchers from …

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Sarepta Enters Gene Therapy Licensing Agreement with Roche Worth Up to $2.85 Billion

CAMBRIDGE, Mass., Dec. 23, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the agreement, Sarepta will …

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FDA Grants Avatrombopag Orphan Drug Designation for the Treatment of Chemotherapy-Induced Thrombocytopenia

STOCKHOLM, Dec. 21, 2019 /PRNewswire/ — Dova Pharmaceuticals, Inc., a wholly owned subsidiary of Swedish Orphan Biovitrum AB (publ) (Sobi™) has been granted Orphan Drug Designation[1] (ODD) by the US Food and Drug Administration (FDA) for avatrombopag for the potential treatment of Chemotherapy-Induced Thrombocytopenia (CIT). Enrollment remains ongoing for the …

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Alnylam’s Lumasiran Met Primary and All Secondary Endpoints in its Phase 3 Study in Patients with Primary Hyperoxaluria Type 1

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), met its primary efficacy endpoint and all tested secondary …

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