GW Pharma’s Epidiolex scores in rare childhood onset epilepsy Phase 3 trial

pharmafile | May 7, 2019 | News story | Manufacturing and Production, Sales and Marketing CBD, Epidiolex, GW Pharma, cannabidiol, cannabis, epilepsy, tsc 

GW Pharma’s Epidiolex has shown positive Phase 3 trial results for the fifth time in a row, after meeting primary endpoints in a randomised, double blind, clinical study of patients with Tuberous Sclerosis Complex (TSC), a rare form of childhood onset epilepsy.

The CBD-based drug reduced seizures by 48.6% and 47.5% from baseline at doses of 25 and 50 mg/kg/day in patients with TSC, compared to a rate of 26.5% for placebo.

“The positive outcome in this trial of EPIDIOLEX in patients with Tuberous Sclerosis Complex expands both our knowledge of this newly available medicine and its potential utility beyond the current indications,” Dr Elizabeth Thiele, Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, Professor of Neurology at Harvard Medical School and the lead investigator of the trial, said.

“The positive results from this trial represent the fifth positive Phase 3 trial for EPIDIOLEX and follows the recent U.S. launch of EPIDIOLEX for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. These new data show EPIDIOLEX reduced TSC-associated seizures, which include both focal and generalized seizures types, expanding the body of reliable science supporting the use of EPIDIOLEX,” GW’s CEO Justin Gover, said.

“With these data, we look forward to submitting an sNDA to the FDA in the fourth quarter with the goal of expanding the product label in 2020 to help the lives of patients suffering with TSC.”

“Some of the most challenging and frustrating aspects of tuberous sclerosis complex (TSC) are seizures that cannot be effectively controlled by existing medications,” explained Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance.

“A new safe and effective treatment option such as Epidiolex is desperately needed.  Further, we are grateful to GW, the researchers and the members of the TSC community who participated in this clinical trial.  We are truly excited about the potential approval of Epidiolex to treat seizures in TSC as it brings much needed hope to those with TSC and their families who live daily with this difficult disorder,” she added.

Shares in the firm were up more than 7% on announcement of the news.

Louis Goss

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