Specialty Pharma Journal Specialty, Precision & Technology
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 …
Tag Archives: Sarepta Therapeutics
December, 2018
June, 2018
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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 …
December, 2017
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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 …
June, 2017
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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 …
January, 2017
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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. …
October, 2016
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5 October
Sarepta Licenses Duchenne Muscular Dystrophy Candidate from Summit Therapeutics
CAMBRIDGE, Mass. and OXFORD, United Kingdom, Oct. 04, 2016 (GLOBE NEWSWIRE) — Sarepta Therapeutics (NASDAQ:SRPT) and Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM) today announced that they have entered into an exclusive license and collaboration agreement granting Sarepta rights in Europe, as well as in Turkey and the Commonwealth of Independent States …
September, 2016
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19 September
Sarepta’s Eteplirsen Wins FDA Approval for Treatment of Duchenne Muscular Dystrophy
The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent …
June, 2016
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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 …
March, 2016
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14 March
Sarepta Announces FDA Advisory Committee Review Date for its DMD Drug Eteplirsen
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen on April 25, 2016. The Prescription Drug User Fee …
January, 2016
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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 …