- Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to certain DMD patients.
- DMD is a rare genetic disease, which mainly affects young men, and is the most common fatal genetic disorder diagnosed in childhood. Because the body does not produce dystrophin in those affected by DMD, muscle tissue does not function properly, the body weakens and eventually the patient is paralyzed and dies. Most patients die before age 30.
- Of the 23,000 boys and young men in Europe with Duchenne Muscular Dystrophy (DMD), about 3,000 will be candidates for BioMarin's treatment if it is approved.
Despite some of the controversy around the issue of designing clinical trials to test DMD drugs in the U.S. and gain approvals, BioMarin is moving forward with its application for drisapersen in Europe. According to BioMarin, approximately 85% of DMD patients in its commercial territories are located outside of the U.S. However, although BioMarin's drug has been validated by the EMA, it still has to go through the full EMA submission process, which shoudl lead to a final approval decision by Q3 2016.