Swiss giant Roche (SWX: ROG) has announced that it has received marketing approval from the US FDA for its Evrysdi (risdiplam) tablet. The tablet is approved to treat spinal muscular atrophy (SMA) patients aged 2 months and above and weighing over 20 kilograms (44 pounds).
Drug Details
Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier. It is the only non-invasive disease-modifying treatment for SMA, a severe, progressive neuromuscular disease that can be fatal. The drug has been registered in over 100 countries and has benefited more than 16, 000 patients worldwide.
Approval and Study Results
The approved 5 mg Evrysdi tablet can be swallowed whole or dispersed in water. In a bioequivalence study, it provided comparable exposure to risdiplam to the original oral solution. Previously, the FDA approved Evrysdi powder for oral solution to treat SMA in adults and children.-Fineline Info & Tech
