Latest news with #DMD
Business Wire
8 hours ago
- Business
- Business Wire
Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). 'Receipt of Orphan Drug Designation from the FDA for PBGENE-DMD underscores the tremendous unmet need and urgency to deliver safe treatments that significantly improve muscle function over time for boys living with Duchenne muscular dystrophy,' said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. 'This regulatory milestone builds on our recent receipt of Rare Pediatric Disease designation and, together with our preclinical body of evidence, gives us tremendous confidence as we move this program towards the clinic. Looking ahead, we remain in active dialogue with the FDA as we advance PBGENE-DMD toward regulatory milestones, with clinical data anticipated in 2026.' The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides sponsors certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the U.S. if the drug is ultimately approved for that use. About PBGENE-DMD PBGENE-DMD is Precision's development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near full-length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address up to 60% of the DMD patient population. In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored the body's ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement. PBGENE-DMD was recently granted FDA Rare Pediatric Disease (RPD) designation for the treatment of DMD. The Company is advancing the final IND-enabling toxicology studies and is working closely with leading DMD clinicians to design a first in human trial optimized for safety and efficacy. Clinical grade material is in preparation, with initial clinical data expected in 2026. About Precision BioSciences, Inc. Precision's two lead programs, PBGENE-HBV, for chronic Hepatitis B, and PBGENE-DMD, for Duchenne muscular dystrophy, are focused on areas with large patient populations with high unmet need. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company's pipeline prioritizes in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our and our partners' and licensees' product candidates and gene editing approaches; the potential of PBGENE-DMD to safely drive meaningful improvement in functional and durable benefit over time for up to 60% of patients with DMD; the design on PBGENE-DMD to permanently edit a patient's own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein proven known to be functional in humans; the benefits of an Orphan Drug Designation including financial incentives and regulatory exclusivity; the potential value of a Priority Review Voucher(if awarded) including priority review for a different product or sale to another sponsor; the expected timing of regulatory processes and clinical operations (including IND and/or CTA filings, studies, enrollment and clinical data for PBGENE-DMD; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as 'aim,' 'anticipate,' 'appear,' 'approach,' 'believe,' , 'confidence', 'contemplate,' 'could,' 'design' 'designed,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look,' 'may,' 'mission,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'pursue,' 'should,' 'strive,' 'suggest,' 'target,' 'will,' 'would,' or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators' or other licensees' ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' or other licensees' development of product candidates; our or our collaborators' or other licensees' ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators' and other licensees' ability to enroll patients; changes in interim 'top-line' and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees' ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators' or other licensees' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption 'Risk Factors' in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at and the Investors page of our website under SEC Filings at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Business Insider
15 hours ago
- Business
- Business Insider
Wainwright Analyst Slaps $0 Price Target on Sarepta Stock (SRPT)
Sarepta Therapeutics (SRPT) is under mounting Wall Street scrutiny amid safety concerns surrounding its flagship gene therapy, Elevidys. In a striking call, analyst Mitchell Kapoor at H.C. Wainwright assigned a $0 price target to SRPT stock, citing the tension between the company and the FDA (Food and Drug Administration). Meanwhile, Kapoor maintained his Sell rating on SRPT stock. 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. SRPT stock has plunged more than 30% over the past five trading days, following Friday's news that the FDA requested Sarepta halt all shipments of Elevidys. In response, the company announced a voluntary pause in U.S. shipments of the therapy. For context, Elevidys is a one-time gene therapy from Sarepta that treats Duchenne muscular dystrophy (DMD) in patients aged four and up. HC Wainwright Sees FDA Clash as 'Unwinnable' Kapoor's rare price target cut comes after Sarepta initially refused to voluntarily pause Elevidys shipments, despite an FDA request following three patient deaths linked to the treatment. Additionally, Kapoor called the conflict with the FDA 'unwinnable' and warned that regulators are likely to force Elevidys off the market. A potential withdrawal could wipe out the therapy's revenue stream, leaving Sarepta stock effectively worthless. He also cautioned investors against single-product biotech companies, warning that such a business model may not be sustainable. Other Analysts Weigh In on SRPT Stock After Friday's decline, more than six analysts have downgraded their ratings on Sarepta stock. Notably, both Deutsche Bank Securities and Needham & Co. issued Sell ratings. Needham's analyst Gil Blum believes the ongoing 'showdown' between the FDA and Sarepta will likely end with formal regulatory action against Elevidys. He also warns that a potential market withdrawal could jeopardize Sarepta's ability to meet its $1.15 billion debt obligations due in 2027. On the other hand, J.P. Morgan's analyst Anupam Rama reiterated his Buy rating on SRPT stock, predicting an upside of over 50%. Rama believes Elevidys will likely remain available for ambulatory patients, as no deaths have been reported in that subgroup to date. These patients can walk independently or with minimal support. What is SRPT's Stock Price Forecast? According to TipRanks, Wall Street has a Hold consensus rating on SRPT stock, based on six Buys, 17 Holds, and three Sells assigned in the last three months. The average Sarepta Therapeutics stock price target of $30.82 implies a 131.4% upside potential.
Business Wire
2 days ago
- Business
- Business Wire
Sarepta Therapeutics Announces Voluntary Pause of ELEVIDYS Shipments in the U.S.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: Today, 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. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Forward-Looking Statements This statement contains 'forward-looking statements.' Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'will,' 'may,' 'intend,' 'prepare,' 'look,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, ELEVIDYS, ongoing interactions with FDA related to ELEVIDYS, and our decision to voluntarily and temporarily pause shipments of ELEVIDYS in the United States. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, the results of future research may not be consistent with past positive results, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading 'Risk Factors' in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us.
Business Wire
2 days ago
- Business
- Business Wire
Rosen Law Firm Urges Capricor Therapeutics, Inc. (NASDAQ: CAPR) Stockholders with Large Losses to Contact the Firm for Information About Their Rights
NEW YORK--(BUSINESS WIRE)--Rosen Law Firm, a global investor rights law firm, announces that a shareholder filed a class action lawsuit on behalf of purchasers and acquirers of Capricor Therapeutics, Inc. (NASDAQ: CAPR) securities between October 9, 2024 and July 10, 2025, both dates inclusive (the 'Class Period'). Capricor is a clinical-stage biotechnology company. For more information, submit a form, email attorney Phillip Kim, or give us a call at 866-767-3653. The Allegations: Rosen Law Firm is Investigating the Allegations that Capricor Therapeutics, Inc. (NASDAQ: CAPR) Misled Investors Regarding its Business Operations. According to the lawsuit, throughout the Class Period, defendants provided investors with material information concerning deramiocel, Capricor's lead cell therapy candidate drug for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Defendants' statements included, among other things, Capricor's ability to obtain a Biologics License Application (BLA) for deramiocel from the U.S. Food and Drug Administration (FDA). Further, defendants provided these overwhelmingly positive statements to investors while, at the same time, disseminating false and misleading statements and/or concealing material adverse facts concerning its four-year safety and efficacy data from its Phase 2 HOPE-2 trial study of deramiocel. The lawsuit alleges this caused shareholders to purchase Capricor's securities at artificially inflated prices. When the true details entered the market, the lawsuit claims that investors suffered damages. What Now: You may be eligible to participate in the class action against Capricor Therapeutics, Inc. Shareholders who want to serve as lead plaintiff for the class must file their motions with the court by September 15, 2025. A lead plaintiff is a representative party who acts on behalf of other class members in directing the litigation. You do not have to participate in the case to be eligible for a recovery. If you choose to take no action, you can remain an absent class member. For more information, click here. All representation is on a contingency fee basis. Shareholders pay no fees or expenses. About Rosen Law Firm: Some law firms issuing releases about this matter do not actually litigate securities class actions. Rosen Law Firm does. Rosen Law Firm is a recognized leader in shareholder rights litigation, dedicated to helping shareholders recover losses, improving corporate governance structures, and holding company executives accountable for their wrongdoing. Since its inception, Rosen Law Firm has obtained over $1 billion for shareholders. Attorney Advertising. Prior results do not guarantee a similar outcome.
Yahoo
2 days ago
- Business
- Yahoo
Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys
The ground shook for Sarepta Therapeutics, Inc. (NASDAQ:SRPT) last week as the U.S. Food and Drug Administration (FDA), alarmed by a third patient death tied to its gene therapy platform, pulled a rare move: requesting a voluntary halt to Elevidys shipments. The company's response? A resounding 'no,' igniting a major regulatory clash with severe implications for patients and investors alike. The upheaval began on Friday when the U.S. Food and Drug Administration (FDA) made a rare and impactful announcement: it had placed Sarepta's investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD) on a clinical hold. This decision stemmed from serious safety concerns, including three patient deaths potentially linked to these products, indicating that study participants faced or would be exposed to an unreasonable and significant risk of illness or injury. In light of these developments, the FDA also formally requested Sarepta to voluntarily halt all shipments of Elevidys, the company's approved gene therapy. However, in a move that set the stage for a major regulatory clash with severe implications for both patients and investors, Sarepta definitively refused this three fatalities, which prompted the FDA's strong action, have resulted from acute liver failure in individuals who had received either Elevidys or an investigational gene therapy utilizing the same AAVrh74 serotype, a viral vector used in gene delivery. Notably, one of these deaths occurred during a clinical trial for LGMD, conducted under an investigational new drug application. Adding to Sarepta's challenges, the FDA further revoked the platform technology designation for the company's AAVrh74 Platform Technology. This designation, typically granted to technologies with broad therapeutic promise that can streamline regulatory review, was rescinded because, given the new safety information, the preliminary evidence was deemed insufficient to demonstrate that the AAVrh74 Platform Technology could be incorporated into or utilized by multiple drugs without adverse effects on safety. Elevidys had received traditional approval in June 2024 for ambulatory Duchenne muscular dystrophy (DMD) patients aged four years and older with a confirmed DMD gene mutation. Previously, in June 2023, it secured accelerated, conditional approval for non-ambulatory DMD patients. However, continued approval for non-ambulatory patients is contingent upon verification of clinical benefit through confirmatory trials. With the emergence of this new safety data, the FDA notified Sarepta that Elevidys's indication should now be restricted solely to ambulatory patients. The FDA is continuing its investigation into the risk of acute liver failure, including severe outcomes such as hospitalization and death, associated with gene therapies employing Sarepta's AAVrh74 Platform Technology, and is prepared to take further regulatory actions as necessary. Despite the FDA's stance, Sarepta issued a statement on Friday affirming its decision to continue shipping Elevidys to the ambulatory patient population. The company asserted that its 'comprehensive scientific interpretation of the data... shows no new or changed safety signals in the ambulant patient population.' Sarepta also expressed its commitment to ongoing discussions and information sharing with the FDA to advance their 'shared purpose of protecting patient safety and informed access to care.' The company clarified that the most recent fatal event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004, which is designed to treat a different condition, LGMD Type 2D. Sarepta emphasized that SRP-9004 is administered at a different dose and manufactured using a distinct process from Elevidys. Furthermore, the LGMD study participant who passed away was not treated with Elevidys, and dosing for the SRP-9004 trial had already concluded at the time of death. Sarepta reported this acute liver failure (ALF) event as a life-threatening case to the FDA on June 20 and subsequently notified the agency of the death on July 3. Looking ahead, Sarepta plans to submit the findings of an expert panel and a proposed protocol to the FDA. They will also discuss a proposal to collect additional data on a modified treatment regimen in a new cohort (Cohort 8) of the ENDEAVOR study (Study SRP-9001-103). This is intended as a pathway to re-establish dosing for non-ambulant patients in the future. Price Action: As of Monday's premarket session, Sarepta's stock was down 6.79% at $13.12, reflecting the ongoing concerns and market reaction to these significant developments. Read Next:Photo via Shutterstock Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? SAREPTA THERAPEUTICS (SRPT): Free Stock Analysis Report This article Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
