EVRYSDI (risdiplam)
Evrysdi treats spinal muscular atrophy (SMA) in both children and adults. It is used for patients whose condition stems from mutations in chromosome 5q, which causes a deficiency in the SMN protein. This medication helps address the underlying protein shortage associated with the disease.
How EVRYSDI Works
This drug works by modifying how the SMN2 gene is processed, specifically by increasing the inclusion of exon 7 in messenger RNA transcripts. This process enables the production of full-length SMN protein in the brain, which is otherwise deficient in patients with this condition. It may also influence the splicing of other genes involved in cell cycle regulation and cell death.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2020-08-07
- Patent Cliff
- 2041
- Revenue
- $548M (Q4-2025)
- Routes
- ORAL
- Dosage Forms
- FOR SOLUTION, TABLET
EVRYSDI Approval History
What EVRYSDI Treats
1 indicationsEVRYSDI is approved for 1 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Spinal Muscular Atrophy
EVRYSDI Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Clinical Trial Registry
16 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05861999 HINALEA 2 | BN44621 2023-505161-81-00 | Ph 4 | recruiting | 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 |
| NCT05861986 HINALEA 1 | BN44620 2023-504508-26-00 | Ph 4 | recruiting | A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy |
| NCT05808764 PUPFISH | BN44619 2023-505602-42-00 | Ph 2 | recruiting | A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy |
| NCT07047144 OPAL | SRK-015-005 | Ph 2 | recruiting | A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy |
| NCT05232929 WeSMA | ML43702 | Ph 4 | active not recruiting | Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) |
| NCT03779334 Rainbowfish results posted | BN40703 2018-002087-12, 2023-506009-20-00 | Ph 2 | active not recruiting | A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy |
| NCT05115110 MANATEE | BN42644 2023-506761-65-00 | Ph 2, Ph 3 | active not recruiting | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy |
| NCT03032172 Jewelfish results posted | BP39054 2016-004184-39, 2023-506739-14-00 | Ph 2 | completed | A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy |
| NCT02913482 FIREFISH results posted | BP39056 2016-000778-40 | Ph 2 | completed | Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy |
| NCT02908685 SUNFISH results posted | BP39055 2016-000750-35 | Ph 2 | completed | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants |
| NCT05522361 RISE | RISE | Ph 4 | active not recruiting | Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen |
| NCT04718181 | BP42066 | Ph 1 | completed | Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants |
| NCT03920865 results posted | BP40995 | Ph 1 | completed | 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 |
| NCT03988907 results posted | BP41361 | Ph 1 | completed | A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants |
| NCT03040635 | NP39625 | Ph 1 | completed | A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants |
| NCT02633709 | BP29840 2015-004605-16 | Ph 1 | completed | A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
EVRYSDI FDA Label Details
Indications & Usage
FDA Label (PDF)EVRYSDI is indicated for the treatment of Spinal Muscular Atrophy.
EVRYSDI Patents & Exclusivity
Patents (7 active)
Exclusivity
Pro Intelligence Preview
Deep insights for EVRYSDI
Revenue Insights
- • Q4-2025: $548M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2041
- • 13 active patents
Trial Analysis
- • 16 total trials
- • Stage: Stable
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment