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Epidiolex: the CBD drug for severe epilepsy

In brief In detail Cannabis News

What is Epidiolex?

Epidiolex (marketed as Epidyolex in Europe) is a pure cannabidiol (CBD)-based anti-epileptic drug developed by British company GW Pharmaceuticals, now a subsidiary of Jazz Pharmaceuticals. It is the first cannabis-derived medicine to receive US FDA approval, in June 2018, and the first CBD-based medicine reimbursed by the French health insurance scheme.

It comes in the form of a drinkable solution containing 100 mg CBD per milliliter, dissolved in sesame oil with strawberry flavoring. The CBD it contains is extracted from legally cultivated cannabis varieties.
by GW Pharmaceuticals, specifically selected for their high cannabidiol and very low THC content.

 

For which diseases?

Epidyolex is indicated for adults and children aged 2 and over in three situations:

Dravet syndrome (DS) SD is a severe, early-onset genetic epilepsy characterized by frequent, prolonged seizures resistant to conventional antiepileptic drugs. DS affects around 1 in 15,000 to 20,000 children.

Lennox-Gastaut syndrome (LGS) epilepsy: a severe form of childhood epilepsy combining several types of seizures with intellectual impairment. Drug resistance is particularly marked.

Tuberous sclerosis of Bourneville (TSB) A rare genetic disease that can cause benign tumors in several organs, including the brain, and lead to severe epilepsy. In this indication, reimbursement is restricted to patients with drug-resistant epilepsy.

In all cases, Epidyolex is prescribed in combination with other antiepileptics, notably clobazam for SD and SLG.

Clinical results

Phase 3 clinical trials have demonstrated that in patients with Dravet or Lennox-Gastaut syndrome, Epidyolex significantly reduces seizure frequency compared with placebo. FDA-approved data show that 47% of treated patients experience at least a 50% reduction in seizures after 12 weeks of treatment - a considerable achievement for conditions where therapeutic options are extremely limited.

The French National Authority for Health (HAS) has recognized Epidyolex as representing «therapeutic progress» in the treatment of these two syndromes in patients aged 2 and over.

Situation in France

Nominative ATU (2017-2019) available in France from 2017 via a temporary authorization for nominative use, in hospital pharmacies only.

European marketing authorization (2019) On September 19, 2019, the European Commission grants a marketing authorization valid throughout the European Union, under the name Epidyolex.

Compassionate access authorization (2019-2022) Epidyolex: pending pricing and reimbursement, Epidyolex is available under the compassionate access scheme.

Repayment (December 2022) Epidyolex obtains its price and reimbursement in France by decree on December 14, 2022 - a historic first for a cannabis-based medicine. The drug is now available in pharmacies and reimbursed at 65% by the French health insurance system.

Price 1,066.74 euros per 100 mL bottle (reimbursed at 65%, i.e. around 373 euros remaining to be paid before mutual insurance).

Prescription Initial annual hospitalization, reserved for specialists in neurology and neuropediatrics. Renewal possible by any doctor.

International approvals

Epidiolex is approved and marketed in over 30 countries, including the United States (FDA, June 2018), all European Union countries (EMA, 2019), the United Kingdom, Canada and Australia. In the United States,
it is also approved for Bourneville tuberous sclerosis in patients aged one year and over.

→ See also: GW Pharmaceuticals, the British company behind Epidiolex and Sativex

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