FDA approves Roche's (RHHBY.US) subsidiary's Evrysdi tablets new drug application for the treatment of patients with spinal muscular atrophy.

Roche's Genentech announced today 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 stated that Evrysdi is the first non-invasive therapeutic drug to change the course of SMA disease. Evrysdi (5 mg) tablets can be swallowed whole or dissolved in water for administration. The press release highlighted that this is the first tablet medication for treating SMA, providing convenience for patients and caregivers, offering them more freedom and independence. The approval of Evrysdi tablets is based on a study of bioequivalence, which showed that whether Evrysdi (5 mg) tablets are swallowed whole or dissolved in non-chlorinated drinking water (such as filtered water), the exposure level of risdiplam in the body is equivalent to the original oral solution. This means that patients taking the tablets can achieve the same established efficacy and safety as with the oral solution of Evrysdi. For patients using other doses of Evrysdi and those who may prefer the oral solution, Evrysdi oral solution will still be available. Evrysdi works by regulating the splicing of the SMN2 gene mRNA, increasing the mRNA levels capable of expressing normal SMN protein, thus alleviating the symptoms of SMA patients.