The FDA has granted approval to the new drug application for Evrysdi, a medication developed by a subsidiary of Roche (RHHBY.US), for the treatment of patients with spinal muscular atrophy.

Genentech, a subsidiary of Roche (RHHBY.US), today announced that the U.S. FDA has approved the New Drug Application (NDA) for Evrysdi (risdiplam) tablets for the treatment of patients with spinal muscular atrophy (SMA). The press release states that Evrysdi is the first non-invasive therapy to change the course of SMA. The Evrysdi (5mg) tablet can be taken whole or dissolved in water. This is the first tablet medication for SMA, providing patients and caregivers with more freedom and independence. The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which showed that the exposure to risdiplam in the body was equivalent when the Evrysdi (5mg) tablet was taken whole or dissolved in non-chlorinated drinking water (e.g., filtered water) compared to the existing oral solution. This means that patients taking the tablet will receive the same established efficacy and safety as the Evrysdi oral solution. The Evrysdi oral solution will remain available for patients taking other doses of Evrysdi and those who may prefer the oral solution. Evrysdi works by modulating the splicing of the SMN2 gene mRNA, increasing the level of mRNA that can express the normal SMN protein, thereby alleviating the symptoms of SMA.