Evrysdi

(Risdiplam)

ApprovedSmall molecule

Evrysdi is a small molecule that works as a SMN2 Splicing Modifier.[2] It is being used across 5 indications, with its most advanced program in Approved.[1]

Evrysdi is one of 39 small molecule programs targeting ALS. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

ALSApproved

Proximal Spinal Muscular AtrophyApproved

AtrophyActive, not recruiting

Muscular Atrophy, SpinalRecruiting

Spinal Muscular AtrophyActive, not recruiting

ALSApproved

Approved

Proximal Spinal Muscular AtrophyApproved

Approved

AtrophyApproved

Active, not recruiting

Muscular Atrophy, SpinalApproved

Recruiting

Spinal Muscular AtrophyApproved

Active, not recruiting

Competition

Competitive Landscape

17 competitors in ALS

#DrugCompanyPhaseTrials

Evrysdi

Roche

Approved

19 2

Spinraza

Biogen

Approved

—3

Tirosint

IInstitut Jerome Lejeune

Approved

1 4

Revia

Teva

Phase 2

2 5

Enablex

AbbVie

Phase 2

1 6

Ranexa

MMenarini International Operations Luxembourg SA

Phase 2

1 7

Iglarlixi

Sanofi

Phase 3

1 8

Salbutamol

GSK

Phase 2

1 9

Buspirone Hydrochloride

Teva

Phase 3

1 10

Balstilimab

AAgenus Inc.

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

ApprovedActive, not recruiting

Spinal Muscular Atrophy

NCT05232929

PreclinicalActive, not recruiting

Spinal Muscular Atrophy (SMA)

NCT05115110

Q1 2027

Phase 2Active, not recruiting

Muscular Atrophy, Spinal

NCT03779334

Q2 2027

Recruiting

Adult

NCT06978985

Q1 2029

Phase 2Recruiting

Spinal Muscular Atrophy

NCT07047144

ApprovedRecruiting

Muscular Atrophy, Spinal

NCT05861999

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy7 trials

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Completed· Roche logo

Roche·Phase 2·NCT02908685·231 enrolled

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Completed· Roche logo

Roche·Phase 2·NCT02913482·62 enrolled

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Completed· Roche logo

Roche·Phase 2·NCT03032172·174 enrolled

Show 4 more trials

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, not recruiting·CClinic for Special Children·Phase 4·NCT05522361·10 enrolled

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Approved for marketing· Roche logo

Roche··NCT04256265

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)

Recruiting·SScholar Rock, Inc.·Phase 2·NCT07047144·52 enrolled

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

Active, not recruiting· Roche logo

Roche·Approved·NCT05232929·403 enrolled

Muscular Atrophy, Spinal4 trials

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

Recruiting· Roche logo

Roche·Approved·NCT05861999·28 enrolled

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

Recruiting· Roche logo

Roche·Approved·NCT05861986·28 enrolled

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

Active, not recruiting· Roche logo

Roche·Phase 2·NCT03779334·26 enrolled

Show 1 more trial

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

Completed· Roche logo

Roche·Phase 2·NCT05808764·11 enrolled

Tics2 trials

A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants

Completed· Roche logo

Roche·Phase 1·NCT03040635·24 enrolled

A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers

Completed· Roche logo

Roche·Phase 1·NCT02633709·33 enrolled

SMA1 trial

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

Completed·BBakri Elsheikh··NCT05219487·18 enrolled

Adult1 trial

Adult SMA Research and Clinical Hub

Recruiting·NNewcastle-upon-Tyne Hospitals NHS Trust··NCT06978985·600 enrolled

4 more indications

Hepatic Impairment1 trial

A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function

Completed· Roche logo

Roche·Phase 1·NCT03920865·26 enrolled

Interaction1 trial

A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants

Completed· Roche logo

Roche·Phase 1·NCT03988907·35 enrolled

ART1 trial

Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

Completed· Roche logo

Roche·Phase 1·NCT04718181·131 enrolled

Spinal Muscular Atrophy (SMA)1 trial

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 recruiting· Roche logo

Roche·Preclinical·NCT05115110·97 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 7, 2020

EVRYSDI

NDA NDA213535

Feb 11, 2025

EVRYSDI

NDA NDA219285

Sources

References

[1]
ClinicalTrials.gov — 19 trial records for Risdiplam. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Risdiplam compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Risdiplam. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA219285). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Evrysdi. U.S. Food & Drug Administration.
[6]
PubMed — 1 publication indexed for Risdiplam. U.S. National Library of Medicine.
[7]
SEC EDGAR — Roche company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: SMN2 Splicing Modifier[2]
Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: Roche

Patent Landscape[5]

Patent Cliff

Feb 8, 2033

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9586955Feb 8, 2033

9969754May 11, 2035

11827646Jan 25, 2036

11938136Nov 8, 2036

12350273Oct 1, 2038

2 more patents

11534444Oct 4, 2038

12122789Apr 15, 2041

Key Publications[6]

Risdiplam in Type 1 Spinal Muscular Atrophy: FIREFISH Part 2 Results

N Engl J MedSep 29, 2022DOI: 10.1056/NEJMoa2206340

Evrysdi

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q2 2027

Q1 2029

Trials by Indication[1]

Spinal Muscular Atrophy7 trials

Muscular Atrophy, Spinal4 trials

Tics2 trials

SMA1 trial

Adult1 trial

Hepatic Impairment1 trial

Interaction1 trial

ART1 trial

Spinal Muscular Atrophy (SMA)1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Key Publications[6]

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