EVRYSDI
PeakNDAORALFOR SOLUTIONPriority Review
Approved
Aug 2020
Lifecycle
Peak
Competitive Pressure
0/100
Data completeness
6/8 — Strong
Priority Review
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 2020.
Drug data last refreshed 10h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EVRYSDI (risdiplam) is an oral small-molecule survival of motor neuron 2 (SMN2) splicing modifier developed by Roche and approved in 2020. It treats spinal muscular atrophy (SMA) by modifying SMN2 pre-mRNA splicing to increase functional SMN protein production. The drug represents a paradigm shift as the first and only oral SMN2 splicing modifier, offering patients a home-based treatment alternative to IV infusions.
$0.132BPart D
PeakGrowing
14.4 years to LOEPeak-stage product with stable commercial infrastructure; career opportunities focus on optimization, market access, and market expansion rather than launch scaling.
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.