Evrysdi
Neurology· 1476 cited findings· 3 source documents
Company: Roche Holding AG →
What the regulatory reviews say
Evrysdi is a drug indicated for the treatment of spinal muscular atrophy (SMA). In Type 1 SMA patient-derived cells, it demonstrated an EC50 of 12 nM for increasing SMN protein levels. Clinical trials showed a median SMN protein increase of >2-fold after 4 weeks of treatment. Key efficacy outcomes included 29.3% of patients sitting without support (BSID-III) and 90.2% achieving an increase of at least 4 points in CHOP-INTEND score from baseline. Additionally, 85.4% of patients were alive without permanent ventilation, and 92.7% were alive overall.
Safety data includes observations from 465 patients with SMA treated with risdiplam for up to 3 years, with a reported mortality rate of 7.3%. The drug exhibited inhibition of COX1 and COX2 by >84%, and inhibition of acetylcholinesterase by >77%. Regulatory terms include a shelf-life of 24 months for the finished product and stability of 64 days for the reconstituted oral solution. Proposed patient enrollment for post-authorization efficacy studies is 300 patients with a minimum follow-up duration of 5 years.
Key figures — cited to the source page
| EC50 for increasing SMN protein levels in Type 1 SMA patient-derived cells | 12 nM | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.24 |
| Median SMN protein increase after 4 weeks | >2 fold | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.82 |
| Proportion of patients sitting without support (BSID-III) | 29.3% | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.132 |
| Proportion of patients achieving increase of at least 4 points in CHOP-INTEND score from baseline | 90.2% | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.132 |
| Proportion of patients alive without permanent ventilation | 85.4% | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.132 |
| Mortality rate | 7.3% | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.126 |
| Shelf-life of finished product | 24 months | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.23 |
| Stability of reconstituted oral solution | 64 days | Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.23 |
Source documents
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Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.