ELEVIDYS, a first gene therapy to treat Duchenne Muscular Dystrophy got an accelerated approval by FDA.

Cambridge: Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, on 22nd June 2023 announced that the U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl). It is a one-time treatment designed for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval based on an increase in ELEVIDYS micro-dystrophin protein expression in skeletal muscle. The approval of this indication may depend on the results of future studies which will confirm the clinical benefits of the drug. ELEVIDYS is contraindicated in patients with deletions in exons 8 and/or 9 of the DMD gene. According to the Muscular Dystrophy Association, in 2019, approximately 1 in 5,000 live male births were affected by Duchenne. It is predicted that every year about 20,000 children are diagnosed with Duchenne globally.

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a gene therapy that is used to treat Duchenne muscular dystrophy (DMD). It is the first and only gene therapy approved for DMD. Also, it works by delivering a functional copy of the DMD gene into muscle cells. This gene codes for the protein dystrophin, which is essential for muscle function. The presence of dystrophin helps to prevent muscle degeneration and improve muscle function. ELEVIDYS is administered as a single intravenous infusion. It is approved for use in ambulatory pediatric patients aged 4 through 5 years with DMD with a confirmed mutation in the DMD gene.

In addition, the FDA approval of ELEVIDYS is a significant event in the cell and gene therapy market. It is the first gene therapy approved to treat a rare disease, and it is likely to have a major impact on the market. The approval of ELEVIDYS is likely to boost investment in the cell and gene therapy market. Investors are likely to be more interested in investing in companies that are developing gene therapies for rare diseases. This is because gene therapies have the potential to provide a cure for rare diseases, which is a major unmet need.

The approval of ELEVIDYS is also likely to increase competition in the DMD therapeutics market. There are currently a number of other gene therapies in development for DMD, and the approval of ELEVIDYS is likely to accelerate the development of these therapies. This is because the approval of ELEVIDYS has shown that gene therapies can be effective in treating DMD.

The approval of ELEVIDYS is an important milestone for the DMD community as this therapy has the potential to improve the lives of patients by slowing disease progression and preserving muscle function. Also, ELEVIDYS is likely to be a major revenue driver for Sarepta Therapeutics. This approval of ELEVIDYS will likely boost the development of other gene therapies developed by several other companies for DMD.

According to the President, and Chief Executive Officer, Sarepta" The approval of ELEVIDYS is a watershed moment for the treatment of Duchenne. ELEVIDYS is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease." Further, Founding President and Chief Executive Officer, Parent Project Muscular Dystrophy, commented: “Today’s decision marks an important moment in gene therapy for patients living with Duchenne. It’s been the lifelong work of so many in the Duchenne community. Our work continues until all patients in our community have access to therapy.”