Drug Development, FDA

3 for 3 in PhIII: GW Pharma racing to the FDA after bagging another late-stage epilepsy success

Extending its winning streak, GW Pharmaceuticals says the second Phase III for Epidiolex in treating Lennox-Gastaut syndrome — a rare form of epilepsy — has also come through with solidly positive efficacy data. And the UK biotech is now planning to hustle ahead with a new drug application at the FDA in the coming months.


Orrin Devinsky, New York University Langone Medical Center

The data from this second study on the company’s lead cannabinoid therapy were even better than the first, with a median 42% drop in monthly seizures, compared to 17% in the placebo group for a 20 mg dose. The 10 mg dose was also a success, with a 37% drop in the seizure rate.

Shares of GW Pharma $GWPH shot up 15% on the latest success, which may well leave the company poised for a market launch.

Back in March the company excited investors with results for the first of four late-stage studies on Epidiolex, with the drug reducing convulsions in children with treatment-resistant Dravet syndrome by a median average of 39% compared to only 13% in the placebo arm. That data helped inspire blockbuster-sized peak sales estimates for this drug.

GW Pharma had a pre-NDA meeting with the FDA last summer, after it racked up the positive Phase III study for Dravet syndrome. Now it plans to hustle along an NDA for both Dravet and LGS at the same time, rather than wait for another round of late-stage data on Dravet syndrome

Stated Orrin Devinsky, M.D., of New York University Langone Medical Center’s Comprehensive Epilepsy Center and principal investigator in the trial:

“The positive outcome in this second trial of Epidiolex in patients with Lennox-Gastaut syndrome demonstrates the effectiveness of this product in this particularly difficult to treat, childhood-onset epilepsy. The data from the Epidiolex Dravet and LGS studies offers the prospect of an FDA-approved CBD medicine that shows both clinically meaningful seizure reduction and a consistent safety and tolerability profile. I believe Epidiolex has the potential to become an important new option within the field of treatment-resistant epilepsy.”

One patient on 10mg/kg Epidiolex discontinued treatment due to an adverse event, investigators said, compared with six patients on 20mg/kg and one patient on placebo.

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