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Drug

Risdiplam

Approved · EMA
Late-stage development

Also known as Evrysdi, Risdiplamum.

RxNorm2390935ChEMBLCHEMBL4297528UNII76RS4S2ET1
6
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase IV Open-Label 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

Clinical trial2026-07-09Results expected Q1 2028 · ClinicalTrials.gov

An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Clinical trial2026-07-09Results posted · ClinicalTrials.gov

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

Clinical trial2026-07-06Results expected Q1 2028 · ClinicalTrials.gov

A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy

Clinical trial2026-06-11Results expected Q4 2026 · ClinicalTrials.gov

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Clinical trial2026-05-04Completed · ClinicalTrials.gov

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

Clinical trial2026-05-01Results expected Q4 2028 · ClinicalTrials.gov

Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

Clinical trial2026-04-20Results expected Q4 2026 · ClinicalTrials.gov

Risdiplam Exchange in Patients With Spinal Muscular Atrophy (SMA) Previously and Exclusively Treated With Nusinersen

Clinical trial2025-12-31Primary completion · ClinicalTrials.gov

An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy

Clinical trial2025-10-01Results posted · ClinicalTrials.gov

A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy

Clinical trial2024-08-07Results posted · ClinicalTrials.gov

A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients

Clinical trial2024-04-24Results posted · ClinicalTrials.gov

Accelerated approval: Evrysdi (EMA)

Regulatory2021-03-26EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Risdiplam
Aliases & brands
EvrysdiRisdiplamum
RxNorm CUI
2390935
ChEMBL ID
CHEMBL4297528
UNII
76RS4S2ET1
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2021-03-26
Latest approval
2021-03-26
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2021-03-26Accelerated approval
Accelerated approval: Evrysdi (EMA)
Indication: Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.… Show full indication

Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. &nbsp;

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

17 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
17
registered trials across all phases
LATEST COMPLETION 2026
6
Active studies
3
Recruiting
5
Late-stage (III+)
10
Completed
1
Discontinued
PHASE DISTRIBUTIONn = 17
Phase 16Phase 26Phase 2 / 31Phase 44

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase IV Open-Label 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

RecruitingPH IVHoffmann-La Rochen=282029

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

RecruitingPH IVHoffmann-La Rochen=282029

Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

Active, not recruitingPH IVGenentech, Inc.n=4032026

Risdiplam Exchange in Patients With Spinal Muscular Atrophy (SMA) Previously and Exclusively Treated With Nusinersen

Unknown statusPH IVClinic for Special Childrenn=102026

A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy

Active, not recruitingPH II/IIIHoffmann-La Rochen=2592026

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

CompletedPH IIHoffmann-La Rochen=112026

An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy

CompletedPH IIHoffmann-La Rochen=1742025

A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy

CompletedPH IIHoffmann-La Rochen=622023

A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients

CompletedPH IIHoffmann-La Rochen=2312023

Risdiplam - A Phase I, Open-Label, Multi-Period Crossover Study to Investigate the Safety, Food Effect, Bioavailability and Bioequivalence of Oral Doses of Two Different Formulations in Healthy Subjects

CompletedPH IHoffmann-La Rochen=1312023

An Open-Label, Single-Dose, Parallel-Group, Two-Part Study to Evaluate the Pharmacokinetics and Safety of Risdiplam in Subjects With Mild or Moderate Hepatic Impairment Compared to Subjects With Normal Hepatic Function

CompletedPH IHoffmann-La Rochen=262020
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.