At the annual congress of the Muscular Dystrophy Association (MDA), Swiss pharma giant Roche (ROG: SIX) has announced new data for its spinal muscular atrophy (SMA) drug Evrysdi (risdiplam).
A daily oral therapy, Evrysdi is a novel survival motor neuron 2 (SMN2) splicing modifier, licensed from PTC Therapeutics (Nasdaq: PTCT).
Roche and PTC inked a major research and licensing deal in SMA in 2011, which included $30 million as an upfront payment and up to $460 million based on milestones, plus double-digit royalties on sales.
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