Roche Holding AG — Evrysdi EMA EPAR public assessment report (EMEA/H/C/005145, 2021)
Roche Holding AG · 2021 · EMA EPAR public assessment report · company profile →
Report summary
The European Medicines Agency (EMA) granted marketing authorization for Roche's Evrysdi (risdiplam) for spinal muscular atrophy (SMA) treatment. Risdiplam is an oral SMN2 splicing modifier, offering systemic distribution and ease of administration. Non-clinical studies confirmed its quality, stability, and mechanism of action, though some toxicity concerns, like retinal degeneration and male germ cell degeneration, were noted. Clinical trials (FIREFISH, SUNFISH) demonstrated risdiplam's efficacy in improving motor function and survival across various SMA types, with greater benefits in younger patients. While generally well-tolerated, adverse events, particularly in Type 1 SMA patients, were observed. Long-term safety, including potential retinal toxicity and male fertility impacts, will be monitored through post-authorization studies.
Key findings — cited to the page
| Grade 3 AE rate (Type 2/3 SMA, BP39055 Only) | 19.19 per 100PY | BP39055 · p.212 |
| Grade 4 AE rate (BP39055 Part 2 Placebo-Controlled Group) | 0.00 per 100PY | BP39055 Part 2 · p.212 |
| Patients with Grade 3 or 4 AE (Type 2 and 3 SMA) | 53 patients | · p.211 |
| Patients with Grade 3-5 AEs (Type 1 SMA) | 35 patients | · p.210 |
| Grade 4 AE rate (Type 2/3 SMA, BP39055 & BP39054) | 1.16 per 100PY | BP39055 & BP39054 · p.212 |
| Grade 4 AE rate (Type 2/3 SMA, BP39054 Only) | 1.34 per 100PY | BP39054 · p.212 |
| Grade 2 AE rate (All SMA) | 124.48 per 100PY | · p.210 |
| Grade 1 AE rate (All SMA) | 418.31 per 100PY | · p.210 |
| Grade 3 AE rate (All SMA) | 26.47 per 100PY | · p.210 |
| Grade 4 AE rate (All SMA) | 4.79 per 100PY | · p.210 |
| Grade 4 AE rate (Type 2/3 SMA, BP39055 Only) | 1.13 per 100PY | BP39055 · p.212 |
| Grade 3 AE rate (Type 2/3 SMA, BP39055 & BP39054) | 21.43 per 100PY | BP39055 & BP39054 · p.212 |
Source: Roche Holding AG ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →