The European Commission has approved Epidyolex (cannabidiol, CBD) as an add-on therapy to clobazam to treat seizures associated with Lennox‑Gastaut syndrome (LGS) and Dravet syndrome in patients 2 and older.
Epidyolex — which is the first CBD product to be approved by the European Medicines Agency (EMA) — is soon expected to be available in all 28 member countries of the European Union, as well as in Norway, Iceland, and Liechtenstein. (Clobazam is marketed under the brand names Onfi and Sympazan in the U.S., and under Frisium and Tapclob, among others, in the EU.)
The European Commission’s decision follows the favorable opinion of the EMA’s Committee for Medicinal Products for Human Use (CHMP). The U.S. Food and Drug Administration approved the treatment, marketed by GW Pharmaceuticals under the brand name Epidiolex, in June 2018.
“The approval of Epidyolex marks a significant milestone, offering patients and their families the first in a new class of epilepsy medicines and the first and only EMA-approved CBD medicine to treat two severe and life-threatening forms of childhood-onset epilepsy,” Justin Gover, CEO of GW, said in a press release.
“We believe patients and physicians deserve access to rigorously tested and evaluated cannabis-based medicines, manufactured to the highest standards and approved by medicines regulators, and we are delighted to be the first to offer this solution to the epilepsy community.”
Epidyolex is a purified and oral form of CBD, the most common non-psychoactive cannabinoid compound in the cannabis plant. CBD is able to mimic natural compounds by acting on brain receptors, which may cause seizures when faulty. In contrast with other cannabis-plant elements, CBD does not induce a “high” sensation.
The decision was supported by clinical evidence collected from three Phase 3 trials (NCT02091375, NCT02224703, and NCT02224560) and the GWPCARE5 open-label extension study (NCT02224573).
The extension study showed that treatment with Epidyolex, in combination with other anti-epileptic medicines, significantly reduced the frequency of convulsive seizures by 38%–44%, and all seizures by 39%–51% in children with LGS or Dravet.
Treatment with Epidyolex also improved overall health and clinical status in approximately 80% of the children at one year of treatment, as assessed with the Subject/Caregiver Global Impression of Change score.
The treatment was well-tolerated, with the most frequent adverse reactions reported being sleepiness, decreased appetite, diarrhea, fever, fatigue, and vomiting. Approximately 29% of trial participants experienced serious adverse events, with 8% deemed to be associated with Epidyolex.
“The approval of cannabidiol oral solution is an important milestone for patients and families whose lives are significantly impacted by these rare, complex and life-long forms of epilepsy,” said Isabella Brambilla, chairwoman of the Dravet Syndrome European Federation.
“We are very happy that patients will now have access to a much-needed, new treatment option, and one routed through a rigorous clinical trials program and licensed by the EMA.”