Viltolarsen is now commercially available in the United States.
Viltolarsen from NS Pharma is the second FDA-approved targeted treatment for patients with Duchenne Muscular Dystrophy (DMD) who have a confirmed mutation in the DMD exon 53 gene. It can be said that it accounts for about 8% of patients with Duchenne.
The FDA has conditionally approved the drug, but final approval is subject to the release of Phase 3 clinical trial results. The test is currently recruiting 74 4- to 7-year-old boys in various countries, including the United States, Canada, Japan and Europe.
Duchenne is the most common type of muscular dystrophy. It is caused by DMD gene mutations that lead to a lack of dystrophin, a protein that helps keep muscle cells healthy. Typically, the first symptoms of the disease are seen in children 3 to 5 years old. Symptoms get worse with age.
One in 3,600 baby boys has Duchenne and it is very rare in women.
Wiltepso is given by intravenous injection of 80 mg / kg body weight. This injection is done once a week by a trained specialist.
Wiltepso has also received legal approval in Japan.