Angelini S p A — Ontozry EMA EPAR public assessment report (EMEA/H/C/005377, 2021)
Angelini S p A · 2021 · EMA EPAR public assessment report
Report summary
The EMA EPAR for Ontozry (cenobamate) details its assessment for adjunctive treatment of focal-onset seizures in adults, leading to a positive marketing authorization opinion in January 2021. Quality aspects, including impurity profiles and stability, were deemed acceptable. Non-clinical studies demonstrated anticonvulsant efficacy, CNS toxicity, and liver changes in animals, with adverse effects on embryo-foetal development at exposures below human therapeutic levels. Pharmacokinetics highlighted drug-drug interactions and metabolism. Clinical efficacy was established in Study C017, showing significant reductions in seizure frequency and higher responder rates compared to placebo. Safety concerns included dose-dependent adverse events like somnolence and dizziness, DRESS, and QT shortening, though a slower titration scheme was proposed to mitigate common adverse events. The CHMP concluded a positive benefit-risk balance.
Key findings — cited to the page
| Patients achieving seizure freedom with initial ASM | 45.7% | · p.10 |
| Patients achieving seizure freedom after failing two ASMs | 4.4% | · p.10 |
| Responder rate (>=50% reduction in seizure frequency) | 40.7 % | YKP3089C017 · United States, rest of world (excluding EU, Australia, NZ, SA) · p.82 |
| Responder rate (>=50% reduction in seizure frequency) | 57.8 % | YKP3089C017 · United States, rest of world (excluding EU, Australia, NZ, SA) · p.82 |
| Patients achieving seizure freedom with second ASM after initial failure | 11.6% | · p.10 |
| Responder rate (>=50% reduction in seizure frequency) | 21.7 % | YKP3089C017 · United States, rest of world (excluding EU, Australia, NZ, SA) · p.82 |
| Marketing Authorisation Application submission date | 9 March 2020 | Ontozry · EU · p.7 |
| CHMP positive opinion date | 28 January 2021 | Ontozry · EU · p.8 |
| Eligibility to centralised procedure agreed by EMA/CHMP | 26 April 2019 | Ontozry · EU · p.7 |
| Procedure start date | 26 March 2020 | Ontozry · EU · p.8 |
| Responder rate (>=50% reduction in seizure frequency) | 60.4 % | YKP3089C017 · United States, rest of world (excluding EU, Australia, NZ, SA) · p.82 |
| Patients achieving seizure freedom after failing three ASMs | 2.1% | · p.10 |
Source: Angelini S p A ↗ · 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 →