sarepta gene therapy fda approval

ET, Presented by studioID and Thermo Fisher Scientific, Webinar This press release contains "forward-looking statements." First Duchenne gene therapy approved by FDA for young children The EMBARK study is expected to serve as the postmarketing confirmatory trial. BLA 125781 . The agency is set to make a final decision by May 29. Let BioPharma Dive's free newsletter keep you informed, straight from your inbox. FDA AdComm Narrowly Supports Approval Of Sarepta's Gene Therapy For Sarepta will likely launch the gene therapy in the fourth quarter of 2023, Schwartz wrote, and he expects revenue of around $110 million before the end of the year. He's also a co-host of "The Readout LOUD" podcast. This uncertainty stemmed from their previous controversial approval experience with eteplirsen (Exondys 51) in 2016. Hollywood actors' strike: How will it hit TV shows and movies? Every Single Store Closing In 2023, https://www.businesswire.com/news/home/20230622454844/en/. Acute serious liver injury has been observed with ELEVIDYS. I recommended monitoring the AdCom meeting for further insights and suggested holding Sarepta shares or potentially increasing holdings if share prices declined solely due to uncertainty. Applicant: Sarepta Therapeutics, Inc. Cellular, Tissue and Gene Therapies Advisory Committee Meeting I emphasized the need to evaluate current data and trials for SRP-9001 independently. Sie knnen Ihre Einstellungen jederzeit ndern, indem Sie auf unseren Websites und Apps auf den Link Datenschutz- und Cookie-Einstellungen oder Datenschutz-Dashboard klicken. If you have an ad-blocker enabled you may be blocked from proceeding. Weitere Informationen darber, wie wir Ihre personenbezogenen Daten nutzen, finden Sie in unserer Datenschutzerklrung und unserer Cookie-Richtlinie. Access unmatched financial data, news and content in a highly-customised workflow experience on desktop, web and mobile. Get the free daily newsletter read by industry experts. The results came the same day Pfizer announced it had dosed the first patient in a Phase 3 trial of its competing Duchenne muscular dystrophy gene therapy. Topics covered: startup launches, funding, IPOs and much more. This rating may be reevaluated if there are substantial changes in enterprise value, significant alterations to the investment outlook, positive confirmatory trial results, or other notable advancements in their pipeline. | 11 a.m. Sarepta Therapeutics shocked Wall . The U.S. health regulator has granted accelerated approval to Sarepta Therapeutics' first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys. Our confirmatory trial, EMBARK, should read out in the fourth quarter of this year, said Doug Ingram, president and chief executive officer, Sarepta. One Duchenne patient's bittersweet hope for new treatment If approved, the potential one-time therapy could change the way patients with the muscle-wasting disease are treated, although the FDA earlier this week said the company did not provide "unambiguous evidence" that it will benefit patients with DMD. ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose gene transfer therapy for intravenous infusion designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. Other treatments include corticosteroids that have side effects if used long-term, such as excess weight gain and osteoporosis. I wrote this article myself, and it expresses my own opinions. During the meeting, advisers voiced concerns about moving forward without more evidence, given mixed results from the midstage trial. A photograph of a Sarepta building in Boston, Mass. ELEVIDYS (delandistrogene moxeparvovec-rokl) is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS, ELEVIDYS is a one-time treatment designed to treat the underlying genetic cause of Duchenne, Sarepta will host a conference call on June 22 at 4:30 p.m. A Top FDA Official Overrules Reviewers' Rejection of Sarepta's DMD Gene This prompted immediate intervention by Peter Marks, one of the top FDA officials and advocate for accelerated approvals of gene therapy. By the end of June, the agency could clear a gene therapy for Duchenne muscular dystrophy and two vaccines for RSV, as well as issue a precedent-setting decision on a closely watched ALS drug. The single-dose treatment is administered by intravenous infusion. Copyright 2023 Dow Jones & Company, Inc. All Rights Reserved. Leveraging innovative technologies and strategic partnerships, they've developed approved Duchenne muscular dystrophy treatments, including EXONDYS 51, VYONDYS 53, and AMONDYS 45, and are pursuing further disease therapies. However, it's important to remember that Elevidys received accelerated approval, implying that the FDA saw an unmet need and believed in the therapy's potential benefits. DMD is estimated to affect one-in-3,500 male births worldwide, according to the National Organization for Rare Disorders, causing progressive muscle failure. Despite the approaching AdCom meeting, I believed that the positive trial results and the ongoing demand for effective treatments for Duchenne muscular dystrophy would have a minimal impact on SRP-9001's chances of approval. It's designed to be taken just once. However, Schwartz and other analysts cautioned that, even if accelerated approval is granted, insurance coverage may not come until after the Phase 3 trial reads out in late 2023. I recommended monitoring the AdCom meeting for further insights and suggested holding Sarepta shares or potentially increasing holdings if share prices declined solely due to uncertainty. The new drug, however, has its limitations it's only . Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing Sarepta Therapeutics' stock has been under some pressure as of late due to the FDA's narrower-than-expected approval for Elevidys, their one-time gene therapy for Duchenne muscular dystrophy (DMD . Continued approval for this indication in this and other age groups will be contingent upon verification of a clinical benefit in confirmatory trials. Thanks for your thoughts. Topics covered: Drug commercialization, pharma salesforces, generic and biosimilar competition, drug advertising and much more. 1 . -- (BUSINESS WIRE)--May 12, 2023-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of SRP-9001 (de. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. Analysts Disclosure: I/we have no stock, option or similar derivative position in any of the companies mentioned, and no plans to initiate any such positions within the next 72 hours. It has demonstrated its ability to navigate regulatory landscapes and leverage scientific advancements to push the boundaries of current treatment paradigms. Dcouvrez comment nous utilisons vos donnes personnelles dans notre Politique de confidentialit et notre Politique relative aux cookies. Sarepta says FDA may limit gene therapy nod to smaller population In April, the drugmaker was cleared to resume dosing patients in that trial. Consistent with the accelerated approval pathway, the company has committed to the completion of a confirmatory trial. Application comes a year earlier than expected, as company says drug reviewers are open to accelerated review based on data from early-stage trials. The FDA approved the therapy only for paediatric patients aged 4-5 years, on the basis of data suggesting that these younger patients may have benefited most. ELEVIDYS is approved under accelerated review based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. There was an error saving your display name. Close more info about Elevidys, a Gene Therapy for Duchenne Muscular Dystrophy, Gets FDA Approval, https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy, https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene. Previously, in January, the biotechnology company disclosed other testing data and broached the idea of discussing a submission with the FDA. Sarepta asks FDA to approve first gene therapy for Duchenne muscular Leveraging my expansive knowledge, I aim to create meticulously researched, highly informative written materials that equip you with the insights you need to navigate the dynamic world of biotech investing. This restricted label might limit the therapy's initial patient pool and market share, which is likely why the market reacted negatively. FDA approval of Sarepta Duchenne genetic therapy gives me hope - STAT The company has a Phase 3 trial underway called EMBARK, that compares SRP-9001 to a placebo, with data due next year. What is aspartame and what do the new WHO rulings mean? At 4:30 p.m. June 22, 2023, Sarepta will host a conference call and webcast to discuss this update. This accelerated approval is based on an increase in ELEVIDYS micro-dystrophin protein expression in skeletal muscle. shocked Wall Street with news that it will submit its treatment for Duchenne muscular dystrophy for accelerated approval by the Food and Drug Administration before finishing a Phase 3 review of the drug next year. The disclosure speeds up by at least a year the timeline for an FDA approval decision. News release. Copyright 2023 Dow Jones & Company, Inc. All Rights Reserved. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. afficher des publicits et des contenus personnaliss en fonction de vos profils de centres dintrt; mesurer lefficacit des publicits et contenus personnaliss; et. Topics covered: Drug commercialization, pharma salesforces, generic and biosimilar competition, drug advertising and much more. Update: FDA Delays Decision on Sarepta's DMD Gene Therapy For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We encourage investors and potential investors to consult our website regularly for important information about us. This article examines Sarepta Therapeutics considering these recent developments. Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. ", "Todays decision marks an important moment in gene therapy for patients living with Duchenne," said Pat Furlong, founding president and chief executive officer, Parent Project Muscular Dystrophy. The gene therapy, called Elevydis, is made by Sarepta Therapeutics. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene In the meantime, Pfizers study has been delayed following a patient death and side effects in others who experienced muscle weakness. The most common adverse reactions in clinical studies were vomiting, nausea, liver function test increased, pyrexia and thrombocytopenia. I'm driven by the pursuit of truth, constantly adjusting my lens as new information comes to light, ensuring my insights are always timely, accurate, and reflective of the current state of affairs. FDA Briefing Document BLA# 125781/00 FDA panel narrowly backs approval of Sarepta's Duchenne gene therapy Advisers voted 8-6 in favor of an accelerated approval despite uncertainty around the treatment's benefit. Accessed June 22, 2023. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene
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