Sarepta tells FDA it won't halt shipments despite patient deaths
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'We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,' FDA Commissioner Marty Makary said in a statement.
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Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects males, though it has faced scrutiny since its clearance in 2023. The one-time treatment received accelerated approval against the recommendations of some FDA scientists who doubted its effectiveness.
Bloomberg
The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, including those who can no longer walk. Previously, it was only available for younger patients who were still walking.
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Sarepta said Friday that its scientific review showed 'no new or changed safety signals' for younger patients with Duchenne's who have earlier stages of the disease. The company said it plans to keep the drug available for those patients.
'We look forward to continued discussions and sharing of information with FDA,' the company said in a statement.
Sarepta halted shipments last month of the therapy for older boys with Duchenne's, which gradually destroys muscle and skeletal strength, resulting in early death. The move followed the deaths of two teenage boys taking the therapy.
The company also confirmed a third death Friday: a 51-year-old patient who was taking an experimental gene therapy in a trial for a different form of muscular dystrophy. Sarepta said it reported the death to the FDA on June 20. The FDA said Friday it placed that trial on hold.
Sarepta noted that the gene therapy involved in the incident uses 'a different dose and is manufactured using a different process,' than Elevidys.
All three patient deaths were linked to liver injury, a side effect noted in Sarepta's prescribing information.
Earlier this week Sarepta announced it would add a bold warning to drug and lay off a third of its employees. The company did not mention the third patient death in its news release or conference call announcing those changes, sparking pointed criticism from Wall Street analysts.
Company shares fell more than 35% Friday to close at $14.07.
Cambridge, Massachusetts-based Sarepta has received FDA approval for three other Duchenne's drugs since 2016, none of which have been confirmed to work. The company has long been criticized for failing to complete several studies needed to secure full FDA approval of its drugs.
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The FDA approval is the latest regulatory milestone for ANZUPGO, following the European Commission (EC) approval in 2024 and several launches internationally, including Germany, Switzerland, the United Kingdom and the United Arab Emirates. *Ends* About ANZUPGO®(delgocitinib) Cream ANZUPGO® (delgocitinib) cream is currently FDA approved in the U.S. as the first and only treatment for chronic hand eczema (CHE). ANZUPGO is also approved in the European Union, United Kingdom, Switzerland and the United Arab Emirates for the treatment of moderate-to-severe chronic hand eczema (CHE) in adults for whom topical corticosteroids are inadequate or not advisable. ANZUPGO cream is also under investigation in other markets. Use of ANZUPGO in combination with other JAK inhibitors or potent immunosuppressants is not recommended by the U.S. FDA.1 ANZUPGO cream is a topical pan-Janus kinase (JAK) inhibitor for the treatment of moderate-to-severe CHE in adults. It inhibits the activation of JAK-STAT signaling, which plays a key role in the pathogenesis of CHE.7 In 2014, LEO Pharma A/S and Japan Tobacco Inc. (JT) entered into a license agreement in which LEO Pharma gained exclusive rights to develop and commercialize delgocitinib for topical use in dermatological indications worldwide, excluding Japan, where JT retains rights. The full U.S. FDA Prescribing Information and Medication Guide are available here: About Chronic Hand Eczema Chronic hand eczema (CHE) is defined as hand eczema (HE) that lasts for three or more months or relapses twice or more within a year.5,8 HE is one of the most common skin disorders of the hands and in a substantial number of patients, it can develop into a chronic condition.9 CHE affects approximately one in ten adults worldwide.2,3 It is a fluctuating disorder characterized by itch and pain, and patients may experience signs such as erythema, scaling, lichenification, hyperkeratosis, vesicles, edema, and fissures on hands and wrists.6 The pathophysiology is characterized by skin barrier dysfunction, inflammation of the skin, and alterations of the skin microbiome.2 CHE has been shown to cause psychological and functional burdens that impact patient quality of life,10,11 with approximately 70% of individuals who live with severe CHE admitting to problems in performing everyday activities.12 Furthermore, careers and earning potential have also been shown to be impacted by the burden of living with CHE.13 About LEO Pharma LEO Pharma is a global leader in medical dermatology. We deliver innovative solutions for skin health, building on a century of experience with breakthrough medicines in healthcare. We are committed to making a fundamental difference in people's lives, and our broad portfolio of treatments serves close to 100 million patients in over 70 countries annually. Headquartered in Denmark, LEO Pharma has a team of 4,000 people worldwide. LEO Pharma is co-owned by majority shareholder the LEO Foundation and, since 2021, Nordic Capital. For more information, visit References ANZUPGO® (delgocitinib) cream. Prescribing Information. FDA. July 2025. Lee GR, Maarouf M, Hendricks AK, Lee DE, Shi VY. Current and emerging therapies for hand eczema. Dermatol Ther. 2019;32(3):e12840. Quaade AS, Simonsen AB, Halling A-S, Thyssen JP, Johansen JD. Prevalence, incidence, and severity of hand eczema in the general population – A systematic review and meta-analysis. Contact Dermatitis. 2021;84:361–374. Tanimoto A, Ogawa Y, Oki C, Kimoto Y, Nozawa K, Amano W, Noji S, Shiozaki M, Matsuo A, Shinozaki Y, Matsushita M. Pharmacological properties of JTE-052: a novel potent JAK inhibitor that suppresses various inflammatory responses in vitro and in vivo. Inflamm Res. 2015;64:41-51. Lynde C, Guenther L, Diepgen TL, et al. Canadian hand dermatitis management guidelines. J Cutan Med Surg. 2010;14(6):267-284. Erratum in: J Cutan Med Surg. 2011 Nov-Dec;15(6):360. Thyssen JP, Schuttelaar MLA, Alfonso JH, et al. Guidelines for diagnosis, prevention, and treatment of hand eczema. Contact Dermatitis. 2022;86(5):357-378. Dubin C, Del Duca E, Guttman-Yassky E. Drugs for the Treatment of Chronic Hand Eczema: Successes and Key Challenges. Ther Clin Risk Manag. 2020;16:1319-1332. Erratum in: Ther Clin Risk Manag. 2021 Mar 18;17:233. Diepgen TL, Andersen KE, Chosidow O, et al. Guidelines for diagnosis, prevention and treatment of hand eczema. J Dtsch Dermatol Ges. 2015;13(1):e1-e22. Bissonnette R, Diepgen TL, Elsner P, et al. Redefining treatment options in chronic hand eczema (CHE). J Eur Acad Dermatol Venereol. 2010;24 Suppl 3:1-20. Grant L, Seiding Larsen L, Burrows K, et al. Development of a Conceptual Model of Chronic Hand Eczema (CHE) Based on Qualitative Interviews with Patients and Expert Dermatologists. Adv Ther. 2020;37(2):692-706. Dalgard FJ, Gieler U, Tomas-Aragones L, et al. The psychological burden of skin diseases: a cross-sectional multicenter study among dermatological out-patients in 13 European countries. J Invest Dermatol. 2015;135(4):984-991. Cortesi PA, Scalone L, Belisari A, et al. Cost and quality of life in patients with severe chronic hand eczema refractory to standard therapy with topical potent corticosteroids. Contact Dermatitis. 2014;70(3):158-168. Voorberg AN, Loman L, Schuttelaar MLA. Prevalence and Severity of Hand Eczema in the Dutch General Population: A Cross-sectional, Questionnaire Study within the Lifelines Cohort Study. Acta Derm Venereol. 2022;102:adv00626. MAT-84121 July 2025 View source version on Disclaimer: The above press release comes to you under an arrangement with Business Wire India. Business Upturn take no editorial responsibility for the same. Ahmedabad Plane Crash Business Wire India, established in 2002, India's premier media distribution company ensures guaranteed media coverage through its network of 30+ cities and top news agencies.
