The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has provided a positive opinion for Epidyolex® (cannabidiol) as a treatment option for TSC-associated epilepsy in people aged two years and older. The update is a major hurdle crossed in the journey towards the medicine being approved and funded in the different UK nations.
When a medicine receives a positive CHMP opinion, the European Commission (EC) then reviews and considers approval for the marketing of the product in all European countries. The EC usually does this within 6-8 weeks of the CHMP’s positive opinion. Following the EC’s decision, it is expected that the groups responsible for approving medicines in the UK nations – including the National Institute of Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) – will share their recommendations.
The EMA and EC are both involved in medicine approval in Europe because the EMA evaluates the benefits and risks of medicines, whilst the EC evaluates the medicine’s suitability to be marketed in Europe.
The CHMP’s positive opinion is based on data from a positive Phase 3 safety and efficacy study. The study met its primary goal to reduce seizure frequency, with seizure reduction of 49% in people taking cannabidiol compared to 24% for placebo. All key secondary goals of the trial further supported the aims of the primary goal. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified.
The TSA will continue to monitor the situation in Europe, as well as the regulatory groups of the UK nations, and will provide further updates as soon as possible. In the meantime, community members may wish to sign up the TSA’s upcoming event ‘Epidyolex and TSC‘, in which Dr Sam Amin will discuss the medicine, and further TSC-related epilepsy medicines, in more detail.
GW Pharmaceuticals press release: https://ir.gwpharm.com/news-releases/news-release-details/gw-pharmaceuticals-receives-positive-chmp-opinion-epidyolexr?
From lab to patient. The journey of a medicine assessed by EMA: https://www.ema.europa.eu/en/from-lab-to-patient-timeline
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