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 2d ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EVRYSDI (risdiplam) is an oral small-molecule survival of motor neuron 2 (SMN2) splicing modifier approved in August 2020 for spinal muscular atrophy (SMA). It works by modifying the splicing of SMN2 pre-mRNA to increase production of full-length SMN protein, addressing the genetic deficiency underlying SMA. This oral formulation represents a significant treatment option for SMA patients across multiple age groups.
$0.132BPart D
Peak14.8 years to LOEProduct is at peak lifecycle stage with $132M in Part D spending (2023), indicating strong market penetration and mature commercial infrastructure supporting mid-to-large sales teams.
Mechanism 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 Jun 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.