NDAORALFOR SOLUTIONPriority Review
Approved
Aug 2020
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
20
Data completeness
7/8 — Comprehensive
Priority Review
EVRYSDI (risdiplam) by Roche is survival of motor neuron 2 splicing modifiers [moa]. Approved for survival of motor neuron 2 splicing modifier [epc]. First approved in 2020.
Drug data last refreshed 20h ago · AI intelligence enriched 3w 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 for the treatment of spinal muscular atrophy (SMA). The drug works by modifying the splicing of SMN2 pre-mRNA to increase production of functional SMN protein, addressing the underlying genetic defect in SMA. Approved by the FDA on August 7, 2020, EVRYSDI represents a disease-modifying treatment option for patients across multiple SMA types and is the first and only oral SMN2 splicing modifier in its class.
$0.1BPart D
14.9 years to LOEMechanism of Action
Survival of Motor Neuron 2 Splicing Modifiers
Pharmacologic Class:
Survival of Motor Neuron 2 Splicing Modifier
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
Started Apr 2026
30 enrolled
Spinal Muscle Atrophy
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
Started Apr 2026
90 enrolled
Spinal Muscular Atrophy
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Started Aug 2024
28 enrolled
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Started May 2024
28 enrolled
Muscular Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Started Apr 2024
10 enrolled
Muscular Atrophy, Spinal
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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.