Type 1 spinal muscular atrophy is a rare, progressive neuromuscular disease that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam is an orally administered, small molecule that modifies SMN2 pre–messenger RNA splicing and increases levels of functional SMN protein.
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- TRADE NAME: Evrysdi (Genentech)
- INDICATIONS: Treatment of spinal muscular atrophy
- CLASS: Survival motor neuron 2 (SMN2)-directed RNA splicing modifier
- HALF-LIFE: approximately 50 hours
- FDA APPROVAL DATE: 08/07/2020
- CLINICALLY IMPORTANT, POTENTIALLY HAZARDOUS INTERACTIONS WITH:
Metformin, Multidrug and toxin extrusion (MATE) 1 and MATE 2-K protein transporters - PREGNANCY: No adequate data on the developmental risk in pregnant women. In animal studies, administration of risdiplam during pregnancy or throughout pregnancy and lactation resulted in adverse effects on development (embryofetal mortality, malformations, decreased fetal body weights, and reproductive impairment in offspring) at or above clinically relevant drug exposures.
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Page last updated 12/08/2025
