Tag Archives: Sarepta

December, 2018

20 December
Sarepta Completes Submission of NDA Seeking Approval of Golodirsen in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53

CAMBRIDGE, Mass., Dec. 20, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those …
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10 August
Sarepta Expands its Presence in Gene Therapy through Investment and License Agreement with Lacerta

CAMBRIDGE, Mass., Aug. 08, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that it has made a strategic investment and entered into a license and option agreement with Lacerta Therapeutics, a gene …
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20 June
Sarepta Announces Positive Preliminary Results from Phase 1/2a Gene Therapy Trial in Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., June 19, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from …
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27 December
Patients Treated with Sarepta’s Eteplirsen Experienced a Reduction in Pulmonary Decline in Duchenne Muscular Dystrophy Study

CAMBRIDGE, Mass., Dec. 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced that the pulmonary function results from eteplirsen-treated Duchenne muscular dystrophy (DMD) patients (N=12) in Study 201/202 compared …
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21 June
Sarepta and Genethon Enter Gene Therapy Research Collaboration for Duchenne Muscular Dystrophy Treatments

CAMBRIDGE, Mass. and EVRY, France, June 21, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, a non-profit R&D organization dedicated to the development of biotherapies for …
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11 January
Sarepta Enters Research and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, today announced it has entered a research and option agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy program. Dr. Jerry Mendell, M.D. and Dr. Louise Rodino-Klapac, Ph.D., are the lead principal investigators of the program. …
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8 June
FDA Requests Dystrophin Data from Sarepta Prior to Making a Decision on Eteplirsen NDA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 6, 2016– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has requested that Sarepta provide dystrophin data, as measured by western blot, from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as …
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26 April
FDA Panel Votes Against Sarepta’s Drug to Treat Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Apr. 25, 2016– Sarepta Therapeutics, Inc.(NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today announced that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee (PCNSC) met to review the new drug application (NDA) for eteplirsen as a treatment for Duchenne muscular dystrophy amenable to exon 51 …
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8 February
Sarepta Therapeutics Receives Notification of PDUFA Extension for its DMD Drug

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Feb. 8, 2016– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable …
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22 January
Weather Delays FDA Advisory Committee Meeting of Sarepta’s DMD Candidate

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jan. 20, 2016– Sarepta Therapeutics, Inc.(NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee meeting scheduled for Friday, January 22 has been postponed by the FDA due to an anticipated severe winter snowstorm …
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