Rare Daily Staff

GW Pharmaceuticals said the European Commission has approved the marketing authorization for Epidyolex for use as adjunctive therapy of seizures associated with Lennox‑Gastaut syndrome or Dravet syndrome, in conjunction with clobazam, for patients 2 years of age and older. The approval paves the way for the launch of the medicine across Europe.

Lennox-Gastaut syndrome (LGS) typically occurs between ages of 3 to 5 years and can be caused by a number of conditions, including brain malformations, severe head injuries, central nervous system infections and genetic neuro-degenerative or metabolic conditions. In up to 30 percent of patients, no cause can be found. Patients with LGS commonly have multiple seizure types including drop and convulsive seizures, which frequently lead to falls and injuries, and non-convulsive seizures. Resistance to anti-epileptic drugs is common in patients with LGS. Most patients with LGS experience some degree of intellectual impairment, as well as developmental delays and aberrant behaviors.

Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic encephalopathy frequently associated with genetic mutations in the sodium channel gene SCN1A. Initial seizures are often body temperature related, severe, and long-lasting. Over time, patients with Dravet syndrome often develop multiple types of seizures, which can be life threatening. Additionally, the majority of patients will develop moderate to severe intellectual and development disabilities and require lifelong supervision and care.

EC approval is based on results from four randomized, controlled phase 3 trials. These studies incorporated data from more than 714 patients with either LGS or Dravet syndrome. When added to other anti-epileptic therapies, Epidyolex significantly reduced the frequency of seizures in patients with LGS and Dravet syndrome. The most common adverse reactions that occurred in patients treated with the medicine were somnolence, decreased appetite, diarrhea, pyrexia, fatigue, and vomiting.

“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,” said Justin Gover, CEO of GW.

Epidyolex is derived from cannabis and is an oral solution that contains highly purified cannabidiol and was first approved by the U.S. Food and Drug Administration in 2018, under the trade name Epidiolex, for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age or older.

The EC decision is valid in all 28 countries of the European Union, alongside Norway, Iceland and Liechtenstein. The medicine had Orphan Drug designation in the EU.

Photo: Justin Gover, CEO of GW

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