Chugai Pharmaceutical Co., Ltd. (TYO: 4519), a subsidiary of Roche, has submitted a product approval application in Japan for delandistrogene moxeparvovec, marketed globally as Elevidys, a gene therapy aimed at treating Duchenne muscular dystrophy (DMD).
The therapy is specifically indicated for ambulatory boys aged 3 to 7 years with DMD who lack deletions in exons 8 and/or 9 of the DMD gene and do not possess pre-existing immunity to AAV rh74. Elevidys is a recombinant gene therapy designed to deliver a gene that leads to the production of Elevidys micro-dystrophin, a truncated protein (138 kDa) compared to the full-length dystrophin protein (427 kDa) found in healthy muscle cells. The treatment is administered as a single intravenous dose.
The filing is supported by data from the EMBARK Phase III clinical trial, which indicated that while Elevidys did not achieve its primary statistical endpoint of improvement over placebo in the North Star Ambulatory Assessment (NSAA), it met all secondary endpoints, including enhancements in time to rise from the floor, 10-meter walk/run times, time to ascend four steps, and reductions in creatine kinase levels. The gene therapy received accelerated approval in the U.S. in June 2023, transitioning to full approval in June 2024.- Flcube.com
