FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy?utm_medium=email&utm_source=govdelivery LEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age: For the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the DMD gene. For the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene (1, 12.2) The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). https://www.fda.gov/vaccines-blood-biologics/tissue-tissue-products/elevidys?utm_medium=email&utm_source=govdelivery