Genentech Gets New Evrysdi Tablet Approval

FDA has approved a Genentech NDA for a new tablet formulation of Evrysdi (risdiplam) for treating people with spinal muscular atrophy (SMA). The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water, according to the company. “The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam,” it says.

Evrysdi is described as a survival motor neuron (SMN) 2 pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron protein deficiency. It is “designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues, as demonstrated in animal models,” Genentech says.