Epidiolex closest to European pharmacies

O Committee for Medicinal Products for Human Use (CHMP), today delivered a favorable opinion recommending the granting of a Marketing Authorization for the medicine Epidyolex, owned by GW Pharma. Although the mechanism of action is not fully understood, the committee recommends the preparation of cannabidiol alone as an adjunctive therapy. 

O Committee for Medicinal Products for Human Use plays a vital role in the authorization of medicines in the European Union (EU) and is responsible for the initial assessment of EU-wide marketing authorization applications. Epidiolex, a pharmaceutical formulation based on cannabidiol (CBD), has passed its preliminary evaluation scheduled for its June 2019 session.

Epidiolex or Epidyolex was the first drug derived from the cannabis plant, approved by the FDA for use in the US. The oral solution containing highly purified, plant-extracted CBD was submitted to the EMA for review on 5 February 2018. The drug is intended for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome in patients aged two years and over and is the first in a new class of antiepileptic drugs with a new mechanism of action, which is not yet fully understood.

The CHMP was based on the results of four randomized controlled phase 3 trials. These studies incorporate data from more than 714 patients with Lennox-Gastaut syndrome (LGS) or Dravet, two forms of epilepsy with high rates of morbidity and mortality, which significantly burden families and caregivers. Many patients with LGS or Dravet syndrome have multiple seizures per day, which puts their mental development at risk as it is mostly diagnosed in children before the first year of life. Despite current treatment with antiepileptic drugs, these severe forms of epilepsy remain highly resistant to treatment.

The formulation evaluated consisted of an oral solution at 100 mg/ml. THE active substance of Epidyolex is CBD, classified by the EMA as an antiepileptic (ATC code: N03AX). Epidiolex reduces neuron hyperactivity as a result of the interaction with CBD, however the exact mechanisms of action are not fully known. The benefits of Epidyolex are its ability to help manage seizures associated with LGS and DS. The most common side effects are drowsiness, decreased appetite, diarrhea, pyrexia, fatigue, and vomiting.