Cannabidiol Cleared for Tuberous Sclerosis Complex in Europe

The European Commission (EC) has approved cannabidiol (Epidyolex) oral solution as adjunctive treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged 2 years and older.

It’s the third indication for the drug in Europe, having originally been approved by the European Medicines Agency in September 2019 as adjunctive therapy for seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome, in conjunction with clobazam, for patients aged 2 years or older.

TSC is a rare genetic disorder that causes epileptic seizures in up to 85% of patients; up to 60% of those patients fail to respond to standard antiepileptic drugs.

In a phase 3 safety and efficacy study, Epidyolex (25 mg/kg/d) reduced seizure frequency (primary endpoint) by 49%, compared with a reduction of 27% with placebo (P = .0009).

“All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified,” the company said in a news release.

“Those suffering from TSC and those involved in their care will welcome this European approval. Given the demonstrated efficacy and safety profile of Epidyolex, this important development could offer a much-needed treatment option for those most in need,” Alexis Arzimanoglou, MD, coordinator, European Reference Network for Rare and Complex Epilepsies and University Hospitals of Lyon, Lyon, France, said in the release.

“This is an exciting moment for patients and their families who are affected by this debilitating disease and may benefit from this treatment. We now look forward to working with each European member state to make Epidyolex available to appropriate patients across Europe as quickly as possible,” said Justin Gover, CEO of GW Pharmaceuticals, the manufacturer.

In the United States, GW’s cannabidiol (Epidiolex) is approved for the treatment of seizures associated with LGS, Dravet syndrome, and TSC for patients aged 1 year or older.

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