Chugai Pharmaceutical Submits Elevidys Gene Therapy for DMD in Japan
Friday, August 16, 2024
Chugai Pharmaceutical has submitted a regulatory application to Japan's Ministry of Health, Labour and Welfare (MHLW) for delandistrogene moxeparvovec, a gene therapy currently in development (also known as SRP-9001, with the overseas name Elevidys).
Delandistrogene moxeparvovec is a gene therapy designed to address the underlying cause of DMD by promoting the expression of a shortened form of dystrophin in skeletal, respiratory, and cardiac muscles. This therapy has already been granted orphan regenerative medical product status in Japan and received approval as the first gene therapy for DMD in the United States in June 2023. An application has also been submitted in the EU as of May 2024.
This therapy is intended to treat Duchenne muscular dystrophy (DMD) in ambulatory boys aged 3 to 7 years who have specific genetic profiles, excluding those with deletions in exons 8 and/or 9 of the DMD gene and those with pre-existing immunity against AAVrh74.
The therapy has been granted orphan regenerative medical product status in Japan and will undergo priority review by the MHLW.
DMD is a rare genetic disorder that causes muscle degeneration and weakness, affecting approximately one in 5,000 boys worldwide. It leads to a progressive loss of motor function, including the ability to walk, and affects lung and heart function. Most patients require full-time care, often provided by parents, who may struggle with their own physical and mental health as a result. Life expectancy for those with DMD is typically around 28 years.
The disease is caused by mutations in the DMD gene, which prevents the production of dystrophin, a key protein that protects muscle fibres during contraction. Without functional dystrophin, muscle cells are easily damaged, leading to the gradual replacement of muscle tissue with fat and scar tissue.
DMD is a severe, progressive muscle-wasting disease that typically begins in early childhood and leads to significant disability and a reduced life expectancy. There is currently no cure. Delandistrogene moxeparvovec aims to address this by providing a one-time treatment that compensates for the lack of dystrophin, the protein missing in individuals with DMD. If approved, it would be the first gene therapy for DMD available in Japan.
The application is based on data from the global Phase III EMBARK study, which tested the efficacy and safety of delandistrogene moxeparvovec in boys aged 4 to 7 with DMD. Although the primary measure of motor function (the North Star Ambulatory Assessment) did not show a statistically significant improvement compared to placebo, the therapy demonstrated meaningful clinical benefits in several secondary measures, including time to rise from the floor and 10-meter walk time. Chugai remains confident in the positive benefit-risk profile of this therapy and is committed to working with regulatory authorities to achieve approval.
Source:chugai-pharm.co.jp/english/
