GW Pharmaceuticals Scores EU First For Cannabinoid Drug
Executive Summary
GW Pharmaceuticals is pressing on with plans to launch its epilepsy treatment Epidyolex in the EU, starting with France and Germany.
GW Pharmaceuticals’ Epidyolex has become the first ever plant-derived cannabinoid medicine to be authorized by the Europe Commission for use across the EU.
The product, approved for use as adjunctive therapy of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS), is seen as a breakthrough for these two rare, severe and difficult-to-treat forms of epilepsy that have high unmet need.
The EU represents a second major market for the Epidyolex, following its US launch in November 2018, where it is known as Epidiolex.
Synthetic compounds which mimic the effects of cannabis (such as Valeant’s Cesamet (nabilone) for nausea and vomiting are approved in Europe, as is GW/Almirall’s Sativex, a cannabis-derived product for spasticity for multiple sclerosis approved through the decentralized procedure.
Epidyolex also stands in contrast to the many non-medicinal cannibidol (CBD) products which have flooded on to the consumer market in recent years, which have little or no evidence to support their use.
The commission’s authorization for the drug follow’s a recommendation in favor of approval by the European Medicines Agency’s drug evaluation committee, the CHMP, in July.
The therapy will be used in conjunction with clobazam, for patients two years of age and older. Its approval will meet a pent-up demand from parents of children with these conditions, who have been desperate to find treatments to relieve the severe symptoms.
Up to 50,000 children and young adults in Europe have one of the two syndromes.
Epidyolex is not the first treatment approved for DS, Biocodex’s Diocomit has been on the market since 2001, but has not achieved significant market penetration.
Slower Approval
Epidyolex’s approval in Europe has been a long time coming, having originally submitted its dossier to the EMA in December 2017 – a 21-month timeline compared with just 10 months taken by the US Food and Drug Administration.
After filing, GW was able to update its file with the EMA, adding data from a second positive Phase III trial in DS, which became available in late 2018.
The final approval is based on results from four Phase III trials, incorporating data from more than 714 patients with either LGS or DS. The most recent of these trials demonstrated that the drug produced a median reduction in convulsive seizures of 49% compared with a reduction on placebo of 27% in DS.
GW says the company expects to launch first in France and Germany as soon as the next few weeks. A launch in the UK is also planned this year, with the company encouraged by its discussions with NICE, despite an initial “no” from the health technology assessment body last month. (Also see "NICE Says Initial No To Epidyolex In England, But GW Pharma Remains Upbeat " - Pink Sheet, 23 Aug, 2019.)
The company says it has plans to progress pricing and reimbursement in a second wave of 10 EU markets in 2020.
“This approval is the culmination of many years of dedication and collaboration between GW, physicians and the epilepsy community,” said Justin Gover, GW’s CEO.
“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.”
The drug is gaining momentum in the US, where it earned $68.4m in sales in the second quarter.
GW Pharmaceuticals has its eye on expanded Epidyolex into other indications, including Tuberous Sclerosis Complex. The company recently presented promising Phase III trial data, which it expects to submit to the FDA by the end of 2019.
Epidyolex was granted an orphan drug designation from the EMA for the treatment of seizures associated with LGS, DS, and tuberous sclerosis complex.
Rivals In CBD-Based Drugs, Dravet Syndrome
GW Pharma is the leader in CBD-based medicines, and in these hard-to-treat forms of epilepsy, but others are looking to join these markets.
Zogenix's Fintepla, (ZX008, a low dose fenfluramine hydrochloride) is the challenger nearest to market in DS, but received a refusal to file letter from the FDA in April. It says it now ready to re-file in the third quarter, and also recently completed enrolment in a Phase III for LGS.
Meanwhile, Kalytera Therapeutics is looking to break into the CBD-based product space. In August it unveiled positive Phase II clinical data for its CBD product for the prevention of acute graft-versus-host disease.