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FDA Grants Regenerative Medicine Advanced Therapy Designation to MB-107 Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency

NEW YORK and MEMPHIS, Tenn., Aug. 22, 2019 (GLOBE NEWSWIRE) — Mustang Bio, Inc. (NASDAQ: MBIO) and St. Jude Children’s Research Hospital have announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted the Regenerative Medicine …

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FDA Grants Fast Track Designation to Cerecor’s CERC-802 for the Treatment of Mannose-Phosphate Isomerase Deficiency

ROCKVILLE, Md., Aug. 21, 2019 (GLOBE NEWSWIRE) — Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for rare and orphan diseases in pediatrics and neurology, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation …

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FDA Accepts Vertex’s NDA for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combo Treatment for Cystic Fibrosis

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The FDA has granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action …

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FDA Rejects Second Sarepta Duchenne Muscular Dystrophy Treatment, Citing Safety Concerns

CAMBRIDGE, Mass., Aug. 19, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced it had received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen …

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FDA Approves Celgene’s Inrebic for Treatment of Patients with a Rare Bone Marrow Disorder

Today, the U.S. Food and Drug Administration approved Inrebic (fedratinib) capsules to treat adult patients with certain types of myelofibrosis. “Prior to today, there was one FDA-approved drug to treat patients with myelofibrosis, a rare bone marrow disorder. Our approval today provides another option for patients,” said Richard Pazdur, M.D., …

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Regeneron Announces Positive Results from its Phase 3 Trial of Evinacumab in Patients with Homozygous Familial Hypercholesterolemia

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive pivotal Phase 3 results for evinacumab, an investigational angiopoietin-like 3 (ANGPTL3) antibody, in patients with homozygous familial hypercholesterolemia (HoFH). Patients with HoFH have severely elevated levels of bad cholesterol (otherwise known as low-density lipoprotein cholesterol, or LDL cholesterol), and often experience early …

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Sarepta Therapeutics Comments on Erroneous Submission to US FDA Adverse Event Reporting System

CAMBRIDGE, Mass., Aug. 08, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, was informed earlier today that an adverse event report was erroneously submitted to the FDA’s adverse event reporting system (FAERs), a post-marketing surveillance database for approved therapies.  Our investigation to date indicates that …

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NORD and C-Path to Launch FDA-Funded Rare Disease Data Analytics Platform

TUCSON, Ariz. and WASHINGTON, Aug. 7, 2019 /PRNewswire/ — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting on Tuesday, September 17 in Bethesda, MD to formally launch development of a new rare disease data and analytics platform. Funded by a cooperative …

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Novartis Responds to FDA Statement on Data Accuracy Issues with its Recently Approved Gene Therapy Zolgensma

Basel, August 6, 2019 – Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma® (onasemnogene abeparvovec-xioi). First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma. The FDA supports the continued marketing and use of Zolgensma for patients …

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FDA Grants Orphan Drug Designation to Stoke Therapeutics’ Investigational Dravet Syndrome Treatment

BEDFORD, Mass.–(BUSINESS WIRE)–Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product candidate, STK-001, an investigational …

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