Gene/Cell Therapy Quarterly Update: June 2023

This quarterly pipeline wrap-up provides a review of newly approved gene and cell therapies, new indications and news of note on gene and cell therapies drugs in the approval process. See other articles for updates on specialty drugs, biosimilar drugs and  traditional drugs. 

New Drug Information

• Elevidys^® (delandistrogene moxeparvovec-rokl): The U.S. Food and Drug Administration (FDA) granted accelerated approval of Sarepta Therapeutics’ Elevidys, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMDElevidys is a one time treatment that addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells. The Phase 1 ENDEAVOR trial, demonstrated an improvement of 3.8 points from baseline on the North Star Ambulatory Assessment (NSAA) at one year, compared to external controls, following a single infusion of Elevidys. The Phase 3 EMBARK study of 120 boys with DMD ages 4 through 7 years, which will assess change from baseline in the NSAA total score after one year compared to placebo, is expected to readout in the latter half of 2023.¹ Sarepta set the price at $3.2 million per one time Elvidys administration.

New Indications

• None

References

1. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene