The gene therapy delandistrogene moxeparvovec-rokl, also known as Elevidys (Sarepta Therapeutics) is indicated to treat Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 to 5 years, and Sarepta recently filed supplemental data with the FDA seeking to expand the labeled indication.
Investigators have come up with recommended clinical guidelines for the administration of delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics), a recombinant adeno-associated virus (rAAV)–based gene transfer therapy indicated to treat Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 to 5 years who have a confirmed mutation in the DMD gene. Their findings appear in a recent issue of Pediatric Neurology,1 and they cover proposed protocols before and after the 1-time infusion.
The gene therapy was approved by the FDA on June 22, 2023,2 and Sarepta recently filed supplemental data with the FDA seeking to expand the labeled indication by removing the age and ambulatory status restrictions.3
“Delandistrogene moxeparvovec is designed to compensate for the absence of functional dystrophin protein in DMD,” the authors wrote, “by delivering a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered dystrophin protein containing key functional domains of the wild-type protein.”
There are 9 recommendations altogether: 5 for the pre-infusion setting and 4 for the postinfusion settings. The investigators fashioned this guidance, meant to support health care professionals initiating the gene therapy, using data on 85 male patients from the Study 101 (NCT03375164; data cutoff, October 17, 2022), Study 102 (NCT03769116; data cutoff, October 3, 2022), and ENDEAVOR (Study 103; NCT04626674; data cutoff, September 19, 2022) trials. These data accounted for 183 patient-years, and their mean follow-up was 2.2 (0.5-4.8) years.
Prior to infusion, the authors recommend performing the following:
After infusion, the authors recommend these actions:
“The safety profile in the delandistrogene moxeparvovec clinical development program has been consistent, monitorable, and manageable,” the study authors concluded. “These practical considerations of treatment are based on available clinical trial data and are intended to aid physicians treating patients with DMD.
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