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Business Wire
11 hours 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
14 hours 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
a day 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.
Newsweek
a day ago
- Business
- Newsweek
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
Based on facts, either observed and verified firsthand by the reporter, or reported and verified from knowledgeable sources. Newsweek AI is in beta. Translations may contain inaccuracies—please refer to the original content. The U.S. Food and Drug Administration (FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments. The agency announced the action on Friday, citing serious safety concerns and "unreasonable and significant risk" to study participants. The FDA also revoked the company's platform technology designation and requested Sarepta voluntarily halt shipments of its approved drug Elevidys, which the company refused to do. Newsweek reached out to Sarepta Therapeutics via email on Sunday after normal business hours for comment. Why It Matters This regulatory intervention represents a significant safety measure in the gene therapy field, affecting both ongoing clinical trials and an already approved treatment for Duchenne muscular dystrophy. The decision could impact treatment options for families dealing with these devastating muscle-wasting diseases. Duchenne muscular dystrophy is a rare and serious genetic condition that worsens over time, leading to weakness and wasting away of the body's muscles. What To Know The three deaths resulted from acute liver failure in patients treated with either Elevidys or investigational therapies using the same AAVrh74 viral vector technology. One fatality occurred during a clinical trial for limb girdle muscular dystrophy treatment. Elevidys is an adeno-associated virus vector-based gene therapy designed to deliver a gene that leads to production of a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body's muscle cells intact. The drug received traditional approval for ambulatory DMD patients aged 4 and older on June 20, 2024, while maintaining accelerated approval for non-ambulatory patients since June 22, 2023. Under accelerated approval, companies must conduct confirmatory studies to verify clinical benefit. Given the new safety concerns, the FDA has notified Sarepta that the drug's indication should be restricted to ambulatory patients only. The FDA revoked the platform technology designation for Sarepta's AAVrh74 Platform Technology because the preliminary evidence is insufficient to demonstrate that the technology has the potential to be incorporated in multiple drugs without adverse safety effects. What People Are Saying FDA Commissioner Marty Makary, M.D., M.P.H.: "Today, we've shown that this FDA takes swift action when patient safety is at risk. We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges." Dr. Vinay Prasad, Director of FDA's Center for Biologics Evaluation and Research: "Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury." FILE - The Food and Drug Administration seal is seen at the Hubert Humphrey Building Auditorium in Washington, Tuesday, April 22, 2025. FILE - The Food and Drug Administration seal is seen at the Hubert Humphrey Building Auditorium in Washington, Tuesday, April 22, 2025. AP Photo/Jose Luis Magana, File What Happens Next The FDA will continue investigating the risk of acute liver failure and other serious outcomes following gene therapies using Sarepta's AAVrh74 platform technology. The agency has committed to taking additional regulatory actions as needed to protect patients. For current Elevidys patients, the FDA is focusing its safety investigation on ambulatory patients while restricting the drug's use in non-ambulatory patients.
News18
2 days ago
- Climate
- News18
Several districts in Bihar on alert as Gangas water level rises
Patna, Jul 20 (PTI) An alert was issued for several districts of Bihar on Sunday for flood-like conditions as the water level of Ganga was rising amid a forecast for more rain in the region, a statement said. Development Commissioner Pratyaya Amrit held a high-level meeting and reviewed the preparedness of the Disaster Management Department (DMD). 'In view of the continuous rise in the water level of the Ganga river and the forecast of heavy rainfall in the catchment areas of Nepal-based Gandak and Kosi rivers, all wings of the department have been instructed to stay on alert mode," a statement issued by the DMD said. 'Additionally, significant rainfall is expected in most parts of Bihar, including the catchment areas of the Sone river. In such a scenario, all districts concerned must remain alert," it said. The DMD said that in case of flood risks, people should be immediately informed through loudspeakers. If water enters localities, prompt action must be taken to move people to safe places, it said, adding that directions have also been issued to be fully prepared for setting up relief camps and community kitchens. The current water discharge at the Kosi barrage is 1,10,845 cusecs, the statement said, 'However, this may increase if heavy rainfall continues in Nepal". The DMD said that at present, all embankments are secure. Flood-fighting materials have been stockpiled at vulnerable points, and regional engineers and contractors have been put on high alert, it said. 'The department is fully prepared to handle any emergency situation," it added. PTI PKD SOM view comments First Published: July 20, 2025, 19:45 IST Disclaimer: Comments reflect users' views, not News18's. Please keep discussions respectful and constructive. Abusive, defamatory, or illegal comments will be removed. News18 may disable any comment at its discretion. By posting, you agree to our Terms of Use and Privacy Policy.
