Jazz — Epidyolex EMA EPAR public assessment report (EMEA/H/C/004675, 2019)
Jazz · 2019 · EMA EPAR public assessment report · company profile →
Report summary
The EMA EPAR for Epidyolex (cannabidiol) details its regulatory assessment and approval for Lennox-Gastaut and Dravet syndromes in patients aged 2+. Quality, non-clinical pharmacology, and safety assessments covered stability, anticonvulsant effects, toxicology, and pharmacokinetics, noting liver/thyroid changes and drug interactions. Clinical trials demonstrated significant seizure reduction, particularly when co-administered with clobazam. Key safety concerns include dose-dependent hepatotoxicity, especially with valproate, and CNS-related adverse events like somnolence. The CHMP concluded a positive benefit-risk balance for adjunctive therapy with clobazam, with manageable hepatotoxicity risks through monitoring.
Key findings — cited to the page
| Increase in irritability | 5.5 % | · p.163 |
| Incidence of aggression and/or irritability in CBD-OS treated population | 9 % | · p.173 |
| Ethanol content in Epidyolex | 10% v/v | Epidyolex · p.14 |
| Drug-resistant epilepsy in LGS | 90% | · p.13 |
| LGS proportion of childhood epilepsies | 1-4% | · p.12 |
| Number of trial sites | 23 | GWEP1332B Part B · Poland, France, United Kingdom, United States · p.105 |
| Truncating mutations in SCN1A | 40% | · p.12 |
| SCN1A abnormalities in Dravet Syndrome | 70% and 80% | · p.12 |
| Cognitive impairment in LGS by 5 years post onset | ≥ 75% | · p.13 |
| All-cause mortality in LGS vs general population | 14 times greater | · p.13 |
| Intellectual impairment severity in DS | 50% | · p.13 |
| Number of France patients randomized in GWEP1414 | 1 | GWEP1414 · France · p.86 |
Source: Jazz ↗ · 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 →