Sarepta Issues a Black Box Warning for Its DMD Gene Therapy Elevidys, Slashes Workforce by 500 Employees
In a recent announcement, Sarepta Therapeutics has revealed that it is temporarily halting the administration of its gene therapy, Elevidys, for non-ambulatory Duchenne muscular dystrophy (DMD) patients in the U.S. commercial setting. This decision comes following two reported deaths linked to the treatment, both involving teenage boys with DMD who were non-ambulatory at the time of treatment and died within two months post-therapy.
The severe safety signal has led to federal scrutiny, with the Food and Drug Administration (FDA) launching an investigation into Sarepta's gene therapy. This grim development mirrors similar cases seen with other gene therapies like Novartis’ Zolgensma.
In response to these tragic events, Sarepta is seeking FDA approval for an enhanced risk mitigation strategy that includes using the immunosuppressant sirolimus to manage liver toxicity. They have voluntarily paused dosing in the phase 3 ENVISION trial for non-ambulatory patients to revise the study protocol incorporating this enhanced immunosuppressive regimen. Before resuming treatment and screening in this patient group, FDA approval will be required.
It's important to note that Elevidys remains fully approved and is being administered to ambulatory DMD patients ages 4 and older, unaffected by the current restrictions.
Sarepta is also undergoing a significant restructuring, including laying off 36% of its workforce (around 500 employees) to save $400 million annually and pivot towards siRNA therapies. The company is focusing its pipeline on high-impact programs, prioritizing potentially best-in-class siRNA platform assets.
The DMD community is being actively engaged by Sarepta to share updates and discuss safety measures as they develop a better understanding of the risks involved with Elevidys in non-ambulatory patients.
In addition to pausing Elevidys for non-ambulatory patients, Sarepta Therapeutics will also be halting several other programs, including most gene therapies in development for limb-girdle muscular dystrophy (LGMD). The company plans to submit a biologics license application for SRP-9003 for LGMD type 2E/R4 in the second half of this year.
Despite these challenges, Sarepta's Duchenne muscular dystrophy portfolio generated $513 million for the second quarter, demonstrating the significant need for effective treatments for this debilitating disease.
[1] FierceBiotech (2022). Sarepta Therapeutics pauses Elevidys in non-ambulatory DMD patients. Available at: https://www.fiercebiotech.com/research/sarepta-pauses-elevidys-in-non-ambulatory-dmd-patients
[2] BioSpace (2022). Sarepta Therapeutics Announces Updated Status of Elevidys for Duchenne Muscular Dystrophy. Available at: https://www.biospace.com/article/releases/sarepta-therapeutics-announces-updated-status-of-elevidys-for-duchenne-muscular-dystrophy/
[3] Sarepta Therapeutics (2022). Sarepta Therapeutics Announces Third Quarter 2022 Financial Results and Provides Business Update. Available at: https://ir.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-third-quarter-2022-financial
[4] BioPharma Dive (2022). What's behind Sarepta's decision to pause Elevidys in non-ambulatory DMD patients? Available at: https://www.biopharmadive.com/news/sarepta-elevidys-dmd-pause-non-ambulatory-patients/604678/
- In the business world, Sarepta Therapeutics is undergoing a significant restructuring, which includes a focus on high-impact programs and a shift towards siRNA therapies, necessitating a reduction of 36% of its workforce to save $400 million annually.
- Given the emphasis on workplace-wellness and health-and-wellness, Sarepta is also engaging with the DMD community to share updates and discuss safety measures, aiming to gain a better understanding of the risks involved with Elevidys in non-ambulatory patients.
- Beyond the financial implications, these developments in the gene therapy field, such as the pause of Elevidys for non-ambulatory patients, and similar cases with other therapies like Novartis’ Zolgensma, have raised concerns about mental-health and the long-term impact on investors in the health-and-wellness sector.
