The U.S. Food and Drug Administration (FDA) has approved Viltepso (viltolarsen) to treat persons with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
This is the second drug now available for this distinct population that accounts for about 8% of the DMD population—the first drug was golodirsen that was approved in December 2019. Both golodirsen and vitlolarsen were given accelerated approvals by the FDA based on preliminary data and both medications will require the completion of larger clinical trials to confirm their safety and efficacy.
The accelerated approval of vitlolarsen was largely based on data showing the drug to increase dystrophin levels from 0.6% of normal baseline to 5.9% of normal after 25 weeks in boys with DMD who had a DMD gene mutation amenable to exon 53 skipping.
DMD is a rare genetic condition characterized by progressive muscle deterioration and weakness due to the absence of dystrophin. DMD is caused by mutations in the DMD gene. The first symptoms are usually seen between three and five years of age and progress over time and most individuals are wheelchair bound by their early teens.
Vitlolarsen is developed by NS Pharma and is administered intravenously once a week. Golodirsen is developed by Sarepta Therapeutics and is also administered intravenously once a week.
To stay updated on the latest drug approvals, sign up for our newsletter at checkrare.com/sign-up-for-our-newsletter/