Roche Holding AG — Evrysdi EMA EPAR variation assessment report (EMEA/H/C/005145, 2021)
Roche Holding AG · 2021 · EMA EPAR variation assessment report · company profile →
Report summary
This report summarizes the EMA's assessment of a variation for Evrysdi (risdiplam), an SMA treatment, introducing new film-coated and dispersible tablet formulations. The assessment confirmed the quality, manufacturing, and stability of the new formulations. Bioequivalence with the existing oral solution was established, and no new safety concerns were identified. The report also included updated European SMA prevalence data. The CHMP recommended approval of the new tablet formulations, citing potential benefits in administration and transport, while maintaining a positive benefit-risk balance for Evrysdi.
Key findings — cited to the page
| Nonserious adverse events of special interest (NSAESIs) | 0 | BP42066 · p.23 |
| Deaths | 0 | BP42066 · p.23 |
| Approval date | March 2021 | Evrysdi · EU · p.20 |
| Number of subjects enrolled | 131 | BP42066 · p.20 |
| Approval date | August 2020 | Evrysdi · United States · p.20 |
| Serious adverse events (SAEs) | 0 | BP42066 · p.23 |
| Study Phase | Phase I | BP42066 · p.15 |
| Discontinuations due to AE | 6 | BP42066 · p.23 |
| SMA prevalence | 2.12 cases per 100,000 | Italy · p.14 |
| Patients received risdiplam (cumulative) | 4,128 | risdiplam · Rest of the world · p.25 |
| Patients received risdiplam (cumulative) | 10,885 | risdiplam · Global · p.25 |
| Patients received risdiplam (cumulative) | 3,597 | risdiplam · European Economic Area · p.25 |
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 →