Roche Set to Compete With Biogen and Novartis in the SMA Market With its First Oral Therapy
The US FDA has recommended the use of Evrysdi (risdiplam), a candidate of Genentech, a Roche company, for spinal muscular atrophy (SMA), an inherited neuromuscular disease resulting in muscle atrophy, in adults and children two months of age and older.
Evrysdi is orally administered and was evaluated in a broad clinical trial program with patients range from birth to 60 years old, including difficult-to-treat populations whose disease had progressed. It is a survival motor neuron-2 (SMN-2) splicing modifier, designed to increase and sustain SMN protein levels thro...
