regulatory
confidence high
sentiment negative
materiality 0.85
CHMP issues negative opinion on Translarna renewal; PTC plans re-examination
PTC THERAPEUTICS, INC.
- CHMP issued negative opinion on renewal of conditional marketing authorization for Translarna (ataluren) for nmDMD.
- Negative opinion follows EC request for re-review; PTC will request re-examination per EMA guidelines.
- Marketing authorization remains in effect; Translarna expected on market through end of 2024 even if opinion upheld.
- CEO Klein: 'STRIDE real-world evidence should not be ignored'; expert panel agreed evidence shows significant efficacy.