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Evrysdi (RISDIPLAM)
Evrysdi works by increasing the production of survival motor neuron protein.
Evrysdi (risdiplam) is a small molecule modality developed by Genentech Inc, currently owned by the same company. It was FDA-approved in 2020 for the treatment of spinal muscular atrophy. Evrysdi is a patented medication with no generic manufacturers available. Key safety considerations include its half-life of 50 hours. As a treatment for spinal muscular atrophy, Evrysdi aims to improve motor function and quality of life for patients.
At a glance
| Generic name | RISDIPLAM |
|---|---|
| Sponsor | Roche |
| Drug class | Survival of Motor Neuron 2 Splicing Modifier [EPC] |
| Modality | Small molecule |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2020 |
| Annual revenue | 2300 |
Mechanism of action
Risdiplam is survival of motor neuron (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 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain.In vitro and in vivo data indicate that risdiplam may cause alternative splicing of additional genes, including FOXM1 and MADD. FOXM1 and MADD are thought to be involved in cell cycle regulation and apoptosis, respectively, and have been identified as possible contributors to adverse effects seen in animals.
Approved indications
- Spinal muscular atrophy
Common side effects
- Fever
- Diarrhea
- Rash
- Upper respiratory tract infection
- Pneumonia
- Constipation
- Vomiting
- Mouth and aphthous ulcers
- Arthralgia
- Urinary tract infection
Key clinical trials
- A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy (PHASE2)
- A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (PHASE2,PHASE3)
- 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 (PHASE4)
- A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy (PHASE2)
- A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (PHASE4)
- 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 (PHASE3)
- Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 (PHASE1,PHASE2)
- Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Evrysdi CI brief — competitive landscape report
- Evrysdi updates RSS · CI watch RSS
- Roche portfolio CI