Latest news with #SRPT)Therapeutics
Business Insider
4 days ago
- Health
- Business Insider
FDA investigating death of child who received Sarepta's Elevidys
The U.S. FDA is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta (SRPT) Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and received voluntary suspension of product distribution as it investigates the safety concerns. Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Business Insider
22-07-2025
- Business
- Business Insider
Sarepta announces voluntary pause of Elevidys shipments in the U.S.
Sarepta (SRPT) Therapeutics issued the following statement: 'Sarepta Therapeutics notified the U.S. Food and Drug Administration (FDA) of its decision to voluntarily and temporarily pause all shipments of ELEVIDYS (delandistrogene moxeparvovec) for Duchenne muscular dystrophy in the United States, effective close of business Tuesday, July 22, 2025. This proactive step will allow Sarepta the necessary time to respond to any requests for information and allow Sarepta and FDA to complete the ELEVIDYS safety labeling supplement process. The Company looks forward to a collaborative, science-driven review process and dialogue with the FDA.' 'As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,' said Doug Ingram, chief executive officer, Sarepta. 'It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the ELEVIDYS label supplement process.' Sarepta remains committed to transparency and patient safety and will continue to provide timely updates to patients, families, healthcare providers, and the broader Duchenne community as additional information becomes available. Elevate Your Investing Strategy:
Business Insider
19-07-2025
- Business
- Business Insider
Sarepta says will continue to ship Elevidys to ambulant population
Sarepta (SRPT) Therapeutics issued a statement which reads in part: 'Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration to voluntarily halt shipment of Elevidys, our gene therapy for Duchenne muscular dystrophy, in the U.S. We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports…Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care. We recognize that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant Limb-Girdle Muscular Dystrophy (LGMD) patient. We also want to clarify that this tragic event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical stage therapy that is intended to treat a different disease (LGMD Type 2D), is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with ELEVIDYS, and the dosing for the SRP-9004 trial had concluded at the time of his death.' Elevate Your Investing Strategy:
Business Insider
26-06-2025
- Business
- Business Insider
Sarepta treatment of limb girdle muscular dystrophy granted orphan status
A treatment of limb girdle muscular dystrophy type 2C/R5 being developed by Sarepta (SRPT) Therapeutics was granted FDA orphan designation, according to a post to the agency's website. Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
