Broad FDA Approval of Gene Therapy Expands Treatment Choices for Duchenne Muscular Dystrophy: Debra Miller & Michael Kelly, PhD

WATCH TIME: 9 minutes

"Our job today is far from done in this disease, and we've improved the whole therapeutic landscape of exon skipping. We need to do exactly the same with gene therapy."

Originally SRP-9001 (Elevidys; Sarepta Therapeutics), otherwise known as delandistrogene moxeparvovec-rokl, received FDA approval under the accelerated approval pathway for patients with Duchenne muscular dystrophy (DMD) in June 2023. More recently, the agency granted the AAV vector-based gene therapy traditional approval for ambulatory patients with DMD. The company announced that this agency decision also includes an accelerated approval for nonambulatory patients, continued approval for which may be contingent upon verification of clinical benefit in a confirmatory trial.¹

Recently, CureDuchenne hosted webinars in English and Spanish for the Duchenne community after the FDA approved the expansion to the label indication for SRP-9001 to include individuals with DMD with a confirmed mutation in the DMD gene who are at least 4 years of age. The webinars were moderated by Michael Kelly, PhD, the chief scientific officer at CureDuchenne, with Diana Castro, MD, a neuromuscular specialist at the Neurology & Neuromuscular Care Center, who shared the latest information and her perspective as a clinician treating patients with this gene therapy.²

Following the webinars, Debra Miller, the CEO and founder of CureDuchenne (which was started 21 years ago after her son was diagnosed with DMD), sat down with NeurologyLive^® in an interview to discuss the main topics covered during the webinar on the new gene therapy for DMD. Miller and Kelly also spoke about how the newly approved gene therapy differs from previous treatments for this disease. Moreover, the duo talked about the anticipated future developments in gene therapy and exon skipping for DMD.

REFERENCES
1. Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. News Release. Sarepta Therapeutics. Published June 20, 2024. Accessed June 20, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys
2. Global Nonprofit CureDuchenne to Host Informational Webinars Following FDA Label Expansion of Sarepta Therapeutics’ Gene Therapy, ELEVIDYS. News Release. CureDuchenne. Published June 21, 2024. Accessed July 3, 2024. https://cureduchenne.org/general/global-nonprofit-cureduchenne-to-host-informational-webinars-following-fda-label-expansion-of-sarepta-therapeutics-gene-therapy-elevidys/