Data completeness
6/8 — Strong
EVRYSDI by Roche is 2 (smn2) splicing modifier designed to treat patients with spinal muscular atrophy (sma) caused by mutations in chromosome 5q that lead to smn protein deficiency. Approved for spinal muscular atrophy (sma) in pediatric. First approved in 2025.
Drug data last refreshed 12h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EVRYSDI (risdiplam) is an oral small-molecule survival motor neuron 2 (SMN2) splicing modifier approved by the FDA in February 2025 for spinal muscular atrophy (SMA). It works by modifying the splicing of SMN2 pre-mRNA to increase production of full-length SMN protein, restoring motor neuron function in patients with SMA.
$0.132BPart D
GrowthGrowing
14.4 years to LOEAs a recently approved growth-stage product, EVRYSDI is building commercial infrastructure and will attract investment in field sales, medical affairs, and market access roles.
Mechanism of Action
2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger…
Indications (1)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.