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Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above

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Sarepta Therapeutics announced an expanded FDA approval for ELEVIDYS (delandistrogene moxeparvovec-rokl) to treat Duchenne muscular dystrophy (DMD) in patients aged 4 and above. The FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. The continued approval for non-ambulatory patients hinges on the results of a confirmatory trial. ELEVIDYS is contraindicated for patients with mutations in exon 8 and/or exon 9 of the DMD gene. Sarepta's ENVISION study aims to verify the clinical benefits of ELEVIDYS. The expansion represents a significant milestone for the Duchenne community and gene therapy.

Positive
  • FDA expanded approval of ELEVIDYS to DMD patients aged 4+.
  • Traditional approval for ambulatory DMD patients.
  • Accelerated approval for non-ambulatory DMD patients.
  • ELEVIDYS targets the root genetic cause of DMD.
Negative
  • ELEVIDYS contraindicated for patients with exon 8/9 deletions in the DMD gene.
  • Potential for serious liver injury and immune-mediated myositis with ELEVIDYS.
  • Non-ambulatory approval contingent on confirmatory trial results.
  • Myocarditis reported as a side effect post-infusion.

The FDA's expanded approval for ELEVIDYS significantly enhances Sarepta's growth potential. Given the dual approvals—traditional and accelerated—the company's revenue streams could see substantial acceleration. The inclusion of non-ambulatory DMD patients under the accelerated approval pathway adds a sizable new patient demographic, potentially expanding the market size considerably.

This news is particularly relevant because gene therapy treatments usually command high prices due to their complexity and unique benefits. Thus, an uptick in revenue is almost certain, which could positively affect Sarepta's stock price in the short term as investors anticipate increased sales. However, investors should note that continued approval for non-ambulatory patients hinges on the outcomes of the ENVISION phase 3 trial. Should these results fall short, it might dampen future revenue projections and stock performance.

In the longer term, successful post-market studies can solidify ELEVIDYS' market position, further boosting investor confidence and potentially leading to a sustainable increase in stock valuation.

Rating: 1

The FDA approval marks a significant milestone in the treatment of Duchenne Muscular Dystrophy (DMD). The expansion to include both ambulatory and non-ambulatory patients means that ELEVIDYS can now be administered to a broader patient base, potentially improving the quality of life for many more individuals. The accelerated approval for non-ambulatory patients is contingent on future clinical benefits verified through ongoing trials, which adds an element of uncertainty but also reflects confidence in the preliminary data.

The gene therapy approach using adeno-associated virus (AAV) to deliver a transgene coding for micro-dystrophin is cutting-edge, offering a more targeted therapy compared to traditional treatments. However, the safety concerns such as immune-mediated myositis and acute liver injury should not be underestimated. Close monitoring and management protocols are essential to mitigate these risks.

For retail investors, understanding these medical intricacies is crucial. While the potential for significant clinical benefits exists, so do the risks related to adverse reactions and regulatory hurdles that may impact long-term viability and profitability.

Rating: 1

The collaboration between Sarepta and Roche, initiated in 2019, underscores the importance of global partnerships in the biotech industry. This alliance means that while Sarepta will manage U.S. operations, Roche will handle international markets, potentially accelerating global adoption and market penetration of ELEVIDYS. Roche's global reach and regulatory expertise could expedite approvals outside the U.S., opening up additional revenue streams for Sarepta.

From a market perspective, this expanded approval strengthens Sarepta's competitive positioning in the gene therapy space. However, the competitive landscape includes other gene therapies in development or awaiting approval, which may impact market share and pricing strategies. Investors should be aware that while the current news is promising, the gene therapy market is dynamic and subject to rapid changes based on scientific breakthroughs and regulatory decisions.

In the short term, the news likely boosts investor sentiment and stock performance. Long-term benefits hinge on successful international expansion and continued positive clinical outcomes, which would solidify Sarepta's leadership in the field.

Rating: 1

FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients

FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne patients

Sarepta will host an investor conference call on June 21, 2024, at 8:30 a.m. ET

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. The FDA granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

“Representing many years of dedicated research, development, investment and creative energy, the expansion of the ELEVIDYS label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, president and chief executive officer, Sarepta. “At this pivotal moment, I want to give warm thanks to Drs. Jerry Mendell and Louise Rodino-Klapac for their dogged, 20-year pursuit of a gene therapy to treat this ruthless and life-robbing disease, to the FDA for following the scientific evidence to speed delivery of a therapy for a life-threatening rare disease to waiting patients, and to the many clinical investigators and courageous Duchenne families who have participated in the multiple studies that led to this important day.”

“Today’s expansion of the ELEVIDYS label represents the culmination of my 50-year pursuit of a treatment for Duchenne patients and, along with my colleague Dr. Louise Rodino-Klapac, a nearly 20-year effort to optimize and develop a gene therapy that could be safely and effectively delivered to muscle,” said Jerry Mendell, M.D., co-inventor of ELEVIDYS and senior advisor, Medical Affairs, Sarepta. “The initial approval of ELEVIDYS was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne. This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.”

Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of ELEVIDYS in patients with Duchenne muscular dystrophy who are non-ambulatory. ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement.

As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world.

Patients and physicians can access more information about ELEVIDYS at www.SareptAssist.com or by calling 1-888-727-3782.

Conference call details

At 8:30 a.m. ET on June 21, 2024, Sarepta will host a conference call and webcast to discuss this update.

The event will be webcast live under the investor relations section of Sarepta’s website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

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.

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 (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X (formerly Twitter), 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 press release 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, business plans, priorities, research and development programs; the potential benefits and risks of ELEVIDYS; and continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial.

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: continued approval for non-ambulatory patients may be contingent upon verification of clinical benefit; 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, as well as the development of our product candidates and our financial and contractual obligations; our dependence on certain manufacturers to produce our products and product candidates, including any inability on our part to accurately anticipate product demand and to secure in a timely manner manufacturing capacity to meet product demand, may impair the availability of product to successfully support various programs; our data may not be sufficient for obtaining regulatory approval; 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; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; 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 of our product candidates are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; we may not be able to execute on our business plans for various reasons, many of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; 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, 2023, and 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 in this press release. 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.

Investor Contact:

Ian Estepan, 617-274-4052

iestepan@sarepta.com

Media Contact:

Tracy Sorrentino, 617-301-8566

tsorrentino@sarepta.com

Source: Sarepta Therapeutics, Inc.

FAQ

What recent FDA approval did Sarepta Therapeutics receive for ELEVIDYS?

The FDA expanded approval for ELEVIDYS to treat Duchenne muscular dystrophy (DMD) in patients aged 4 and above.

What types of approval were granted to ELEVIDYS by the FDA?

ELEVIDYS received traditional approval for ambulatory DMD patients and accelerated approval for non-ambulatory DMD patients.

What are the risks associated with ELEVIDYS for DMD patients?

Risks include serious liver injury, immune-mediated myositis, and myocarditis. ELEVIDYS is contraindicated for patients with exon 8/9 deletions in the DMD gene.

What are the next steps for Sarepta regarding ELEVIDYS?

Sarepta will conduct the ENVISION study, a phase 3 trial, to verify and confirm the clinical benefit of ELEVIDYS for non-ambulatory DMD patients.

Sarepta Therapeutics, Inc.

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