Myocarditis

A panel of experts who decide the national immunization schedule will meet on Tuesday for the first time in 2025, providing an opportunity for the nation’s top vaccine scientists to publicly discuss the value of products that the new administration has cast doubt on. The CDC’s Advisory Committee on Immunization Practices meeting was postponed from February and is one of three scheduled for this year. The group is expected to discuss a number of vaccines, including shots protecting against the flu, Covid-19, RSV and meningitis. The group will vote on updated recommendations for chikungunya vaccines, meningococcal vaccines for teens and young adults, and RSV vaccination for adults. The administration’s scrutiny of these routine meetings reflects how much anti-vaccine sentiment has risen in recent years, and how HHS Secretary Robert F. Kennedy Jr. stands to reinforce those concerns. “I’m just praying that there will be an ACIP meeting,” Laura Riley, a former ACIP member and chair of obstetrics and gynecology at Weill Cornell Medicine, said in an interview on Monday. A spokesperson for HHS did not immediately respond when asked about Kennedy’s views of the committee and whether he plans to replace members in the future. Vaccine policy in the US is effectively divided into two parts. The FDA and its own advisory group review and decide what vaccines to approve. The CDC and ACIP recommend which Americans should get the approved vaccines. Recommended vaccines are usually covered by insurance. The decisions that ACIP makes, which the CDC director ultimately has final say over, are based on a number of factors, primarily the safety and efficacy of the vaccine. But the committee also considers the cost-effectiveness of the vaccine and the ethics of how certain recommendations, or lack thereof, will impact uptake. The committee reviews vaccine safety data post-licensure, and considers them when updating recommendations. “Unfortunately, there are so much data out there, and people are not aware of how to interpret it, which is why you need an advisory committee like ACIP poring through all of the data in order to make informed decisions,” said Henry Bernstein, a pediatrics professor at the Zucker School of Medicine at Hofstra University and another former ACIP member who served from 2017 to 2021. Not in recent memory has ACIP existed under an administration that so frequently questions the benefits of vaccination. Kennedy leads a separate MAHA Commission that he tasked with investigating the childhood vaccine schedule. He’s continuously questioned whether vaccines are the source of rising autism rates, and has initiated plans for the CDC to study just that, despite numerous studies concluding they do not. Specific kinds of vaccines, including mRNA-based and single-antigen vaccines, have been singled out by either Kennedy or key voices in the MAHA movement he leads. Riley said that acceptance of the committee’s work boils down to effective communication, which she says could be improved. “I don’t think people understand the benefits of science period,” Riley said. “I don’t think that we have communicated clearly enough how important scientific discovery is.” She pointed to the Covid pandemic as a time when the committee could have more directly explained what it did and didn’t know, given how swiftly new data about the vaccines and the virus were being produced. “I don’t think that it was clear to people that we didn’t know everything,” she said, adding that it created a vacuum for alternative sources of information to seep in. Sometimes the decisions themselves were polarizing, like when the committee decided in June 2021 that the benefits of the two-shot mRNA Covid vaccine regimen in men outweighed the risk of vaccine-related heart inflammation known as myocarditis. Kennedy has also questioned whether federal vaccine advisors have a too-cozy relationship with industry. Politico previously reported he was considering replacing some members because of their perceived conflicts. Former ACIP members dismissed the concern, saying the required conflict of interest submissions before each meeting were rigorous and that members frequently erred on the side of caution. Arthur Reingold, a former committee member and an epidemiology professor at the University of California, Berkeley, told Endpoints News that he was barred from voting on a vaccine against HPV because his wife was the director of the CDC’s division of sexually transmitted diseases at the time. “So if people are saying that all of that was basically hiding things or not fully transparent, I don’t know where that accusation comes from, frankly, or what it’s based on,” he said. The CDC now publishes past conflict of interest disclosures. Moving forward, Bernstein hopes there isn’t “dramatic change” to the group, calling its work “incredibly successful.” “What’s being done is critically important, but if we don’t communicate and educate the public at all levels around these vaccines, it’s going to be problematic,” he said. Editor’s note: This story was updated to include the full titles of Henry Bernstein and Arthur Reingold.

Based on the Phase 3 CheckMate-8HW trial, Opdivo plus Yervoy demonstrated reduction in the risk of disease progression or death by 79% vs. chemotherapy in the first-line setting and by 38% vs. Opdivo monotherapy across all lines of therapy 1 The approval was granted more than two months ahead of the Prescription Drug User Fee Act goal date PRINCETON, NJ, USA I April 08, 2025 I Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo ® (nivolumab) plus Yervoy ® (ipilimumab) as a first-line treatment of adult and pediatric patients (12 years and older) with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC). 1 This approval is based on the CheckMate-8HW trial, which is the largest Phase 3 trial (n=839) of immunotherapy in patients with MSI-H/dMMR mCRC, evaluating Opdivo plus Yervoy (n=354) vs. Opdivo monotherapy (n=353) in the all-lines setting and Opdivo plus Yervoy (n=202) vs. investigator’s choice chemotherapy (n=101) (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) in the first-line setting. 1 Opdivo plus Yervoy met the dual primary endpoints of progression free survival (PFS) when compared to Opdivo monotherapy across all lines of therapy and when compared to chemotherapy in the first-line setting, as assessed by Blinded Independent Central Review (BICR). 1 This approval, granted more than two months ahead of the June 23, 2025 Prescription Drug User Fee Act goal date, follows the FDA’s prior decision to grant the application Breakthrough Therapy Designation and Priority Review status. “There is an unmet need for additional treatment options such as a dual immunotherapy approach for patients with previously untreated MSI-H/dMMR unresectable or metastatic CRC, which is an aggressive form of cancer and can be particularly difficult to treat,” said Heinz-Josef Lenz, MD, CheckMate-8HW investigator and Deputy Director for Research Programs and Head of the Gastrointestinal Cancers Program at the USC Norris Comprehensive Cancer Center. 2,3,4,5,6 “The meaningful outcomes in CheckMate-8HW underscore how initiating treatment with the dual immunotherapy combination of nivolumab plus ipilimumab may result in a notable survival benefit. 1,5 This approval has the potential to redefine traditional approaches of care for patients with this form of CRC.” In the CheckMate-8HW trial, Opdivo plus Yervoy demonstrated a 38% reduction in the risk of disease progression or death vs. Opdivo monotherapy in immunotherapy-naïve patients across all lines of therapy (Hazard Ratio [HR] 0.62; 95% Confidence Interval [CI] 0.48–0.81; P =0.0003). 1 Assessing the dual primary endpoint of PFS, the trial demonstrated that median PFS was not reached with Opdivo plus Yervoy (95% CI: 53.8-Not Estimable [NE]) and was 39.3 months with Opdivo monotherapy (95% CI: 22.1-NE). 1 PFS rates at 12-, 24-, and 36-months were also numerically higher compared to Opdivo monotherapy (76% vs. 63%, 71% vs. 56%, and 68% vs. 51%, respectively). 1 In Kaplan-Meier (KM) curves showing PFS rates with Opdivo plus Yervoy compared to Opdivo monotherapy, an early separation was observed at two months and sustained at three years. 1 Opdivo plus Yervoy also met a key secondary endpoint, demonstrating superior overall response rate (ORR) by BICR compared to Opdivo monotherapy (n=296, 71% vs. n=286, 58%; P =0.0011). 1 Of the most common all-cause adverse reactions (ARs) occurring in ≥10% of patients, similar rates of grade 3-4 ARs were observed between Opdivo plus Yervoy and Opdivo monotherapy. 1 The safety profile for the dual immunotherapy combination remained consistent with previously reported data and the ARs observed were manageable with established protocols, with no new safety signals identified. 1,5 Additional safety information can be found in the U.S. Full Prescribing Information for Opdivo . The Opdivo plus Yervoy vs. chemotherapy arm of the CheckMate-8HW trial showed that the combination regimen reduced the risk of cancer progression or death by 79% compared to chemotherapy in first-line patients (HR 0.21; 95% CI: 0.14-0.32; P <0.0001). 1 This arm also assessed the other dual primary endpoint of PFS, where median PFS was not reached with Opdivo plus Yervoy (95% CI: 38.4-NE) compared to 5.8 months with chemotherapy (95% CI: 4.4-7.8). PFS rates were numerically higher with Opdivo plus Yervoy vs. chemotherapy at 12- and 24-months (79% vs. 21% and 72% vs. 14%, respectively). 1 KM curves comparing PFS with Opdivo plus Yervoy vs. chemotherapy showed an early separation at three months, which was sustained through two years. 1 The regimen of Opdivo plus Yervoy represents the first-ever dual immune checkpoint inhibitor combination to demonstrate significant efficacy benefit compared to Opdivo monotherapy and chemotherapy in MSI-H/dMMR mCRC patients. 1,5 Opdivo and Yervoy are associated with the following Warnings and Precautions: severe and fatal immune-mediated adverse reactions including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, nephritis with renal dysfunction, dermatologic adverse reactions, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials. 1 Please see the Important Safety Information section below. “This approval marks our ninth indication for an Opdivo -based treatment in the gastrointestinal space. 1 We are witnessing the transformative potential of dual immunotherapy in treating GI cancers,” said Wendy Short Bartie, senior vice president of Oncology Commercialization at Bristol Myers Squibb. 2,3,4,5 “People with MSI-H/dMMR metastatic colorectal cancer face high unmet need, and Opdivo plus Yervoy is an important new approach in the first-line setting. 2,3,4,5 This milestone can offer hope, and it underscores our commitment to continue reaching more patients with new treatment options.” 1 “Colorectal cancer is the third most commonly diagnosed cancer and the second most common cause of cancer-related death for men and women combined in the U.S., and concerning trends show that incidence is increasing in people younger than 50,” said Nicole Sheahan, President of the Global Colon Cancer Association. 6,7 “Despite the prevalence of CRC, there remains a high unmet need, highlighting the urgency for additional treatment options. 2,3,5,6,7 We are thrilled with this FDA approval as Opdivo plus Yervoy offers an exciting new first-line approach for patients with MSI-H/dMMR metastatic colorectal cancer.” 1 Opdivo as a single agent, or in combination with Yervoy , was previously granted accelerated approval in MSI-H/dMMR CRC adult and pediatric patients (12 years and older) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. 1 Today’s FDA decision converts this second-line indication to full approval for Opdivo monotherapy and expands the indication for Opdivo plus Yervoy into the first-line setting based on the CheckMate-8HW trial. 1 About CheckMate-8HW CheckMate-8HW is a Phase 3, randomized, multicenter, open-label trial evaluating Opdivo plus Yervoy compared to Opdivo alone or chemotherapy (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) in patients with unresectable MSI-H/dMMR mCRC. 8 In the CheckMate-8HW study, 839 patients were randomized to receive either Opdivo monotherapy ( Opdivo 240 mg Q2W for six doses, followed by Opdivo 480 mg Q4W), Opdivo plus Yervoy ( Opdivo 240 mg plus Yervoy 1 mg/kg Q3W for four doses, followed by Opdivo 480 mg Q4W), or investigator’s choice of chemotherapy. 8 The dual primary endpoints of the trial were PFS for Opdivo plus Yervoy compared to Opdivo alone across all lines of therapy and PFS for Opdivo plus Yervoy compared to chemotherapy in the first-line setting, as assessed by Blinded Independent Central Review (BICR). 8 The study is ongoing to assess various secondary endpoints, including overall survival (OS), and BMS will continue to work with the study investigators to present these data and longer-term follow-up in the future. 8 Select Safety Profile from CheckMate-8HW The safety analysis in CheckMate-8HW included 288 patients, of whom 200 received Opdivo plus Yervoy. 1 Serious adverse reactions occurred in 46% of patients receiving Opdivo plus Yervoy . 1 The most frequent serious adverse reactions reported in ≥1% of patients who received Opdivo plus Yervoy were adrenal insufficiency (2.8%), hypophysitis (2.8%), diarrhea (2.0%), abdominal pain (2.0%), small intestinal obstruction (2.0%), pneumonia (1.7%), acute kidney injury (1.4%), immune mediated enterocolitis (1.4%), pneumonitis (1.4%), colitis (1.1%), large intestinal obstruction (1.1%), and urinary tract infection (1.1%). 1 The most common adverse reactions reported in ≥ 20% of patients treated with Opdivo plus Yervoy were fatigue, diarrhea, pruritis, abdominal pain, musculoskeletal pain, and nausea. 1 Fatal adverse reactions occurred in 2 (0.6%) patients who received Opdivo plus Yervoy ; these included myocarditis and pneumonitis, 1 each. 1 Opdivo and/or Yervoy were discontinued in 19% of patients and were delayed in 48% of patients for an adverse reaction. 1 About Colorectal Cancer Colorectal cancer (CRC) is cancer that develops in the colon or the rectum, which are part of the body’s digestive or gastrointestinal system. 9 With more than 154,000 new cases estimated to be diagnosed in the United States in 2025, CRC is the third most commonly diagnosed cancer. 7,10 Trends show that incidence is increasing in people younger than 50, and mortality rates have increased in people younger than 55 since the mid-2000s. 7 Mismatch repair deficiency (dMMR) occurs when the proteins that repair mismatch errors in DNA replication are missing or non-functional, leading to microsatellite instability-high (MSI-H) tumors. 11,12 Up to 7% of people with mCRC have MSI-H/dMMR tumors and may often have poor outcomes with standard chemotherapy. 5 INDICATIONS OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC). OPDIVO ® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy. OPDIVO ® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT). OPDIVO ® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC). OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC). OPDIVO ® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma. Please see U.S. Full Prescribing Information for OPDIVO and YERVOY . Clinical Trials and Patient Populations CheckMate-649–previously untreated advanced or metastatic gastric cancer, gastroesophageal junction and esophageal adenocarcinoma; CheckMate-577–adjuvant treatment of esophageal or gastroesophageal junction cancer; 8HW: Previously CheckMate-142–MSI-H or dMMR metastatic colorectal cancer in combination with YERVOY; 8HW: Previously CheckMate-142–MSI-H or dMMR metastatic colorectal cancer, as a single agent; Attraction-3–esophageal squamous cell carcinoma; CheckMate-648—previously untreated, unresectable advanced recurrent or metastatic esophageal squamous cell carcinoma in combination with chemotherapy; CheckMate-648—previously untreated, unresectable advanced recurrent or metastatic esophageal squamous cell carcinoma combination with YERVOY; CheckMate-040–hepatocellular carcinoma, in combination with YERVOY. Bristol Myers Squibb: Creating a Better Future for People with Cancer Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine and, through innovative digital platforms, are turning data into insights that sharpen their focus. Deep understanding of causal human biology, cutting-edge capabilities and differentiated research platforms uniquely position the company to approach cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. As a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future. About Bristol Myers Squibb’s Patient Access Support Bristol Myers Squibb remains committed to providing assistance so that cancer patients who need our medicines can access them and expedite time to therapy. BMS Access Support ® , the Bristol Myers Squibb patient access and reimbursement program, is designed to help appropriate patients initiate and maintain access to BMS medicines during their treatment journey. BMS Access Support offers benefit investigation, prior authorization assistance, as well as co-pay assistance for eligible, commercially insured patients. More information about our access and reimbursement support can be obtained by calling BMS Access Support at 1-800-861-0048 or by visiting www.bmsaccesssupport.com . About the Bristol Myers Squibb and Ono Pharmaceutical Collaboration In 2011, through a collaboration agreement with Ono Pharmaceutical Co., Bristol Myers Squibb expanded its territorial rights to develop and commercialize Opdivo globally, except in Japan, South Korea and Taiwan, where Ono had retained all rights to the compound at the time. On July 23, 2014, Ono and Bristol Myers Squibb further expanded the companies’ strategic collaboration agreement to jointly develop and commercialize multiple immunotherapies – as single agents and combination regimens – for patients with cancer in Japan, South Korea and Taiwan. About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn , X , YouTube , Facebook and Instagram . References SOURCE: Bristol Myers Squibb

« Back 04/04/25 8:44 AM EDT CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 4, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy. Following the safety update on acute liver failure that was issued on March 18, European Union (EU) reference member country authorities requested that the independent data monitoring committee (DMC) meet to review the adverse event. While the analysis is being finalized, recruitment and dosing in certain clinical studies of ELEVIDYS are temporarily halted. The independent DMC met on April 3 and concurred that based on the totality of evidence, the overall benefit-risk profile remains favorable to continue dosing in the paused clinical trials without changes to the study protocols. At the request of EU regulators, Sarepta and Roche will submit this information in a response to the temporary halt within a week. Evaluation of the submission and the subsequent decision for lifting the temporary halt will follow the EU regulatory process. The clinical studies affected by the temporary halt are Study SRP-9001-302 (ENVOL), Study SRP-9001-303 (ENVISION) and Study SRP-9001-104. Monitoring and data collection for already-enrolled participants continues, and we do not anticipate a material impact on the timeline for these studies. 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 (noted hereafter as “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 www.fda.gov/medwatch 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 leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. 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, research and development programs, the potential timing of clinical trials and the potential benefits and risks of ELEVIDYS. 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: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; 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; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on our business; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20250404491162/en/ Investor Contact: Ian Estepan 617-274-4052 iestepan@sarepta.com Media Contacts: Tracy Sorrentino 617-301-8566 tsorrentino@sarepta.com Kara Hoeger 617-710-3898 KHoeger@sarepta.com Source: Sarepta Therapeutics, Inc.