WASHINGTON (AP) — A medicine made from the marijuana plant moved one step closer to U.S. approval Thursday after federal health advisers endorsed it for the treatment of severe seizures in children with epilepsy.
If the Food and Drug Administration follows the group’s recommendation, GW Pharmaceuticals’ syrup would become the first drug derived from the cannabis plant to win federal approval in the U.S.
The 13-member FDA panel voted unanimously in favor of the experimental medication made from a chemical found in cannabis — one that does not get users high. The panelists backed the drug based on three studies showing that it significantly reduced seizures in children with two rare forms of childhood epilepsy.
“This is clearly a breakthrough drug for an awful disease,” said panel member Dr. John Mendelson, of the Friends Research Institute in Baltimore, Maryland.
A University of Cincinnati scientist who served as lead researcher in a clinical trial testing Epidiolex said the drug delivered “impressive” results among 255 patients nationwide who suffer more than 50 seizures a month from severe forms of epilepsy.
“These are pretty impressive numbers,” Dr. Michael Privitera said. “It’s pretty uncommon that we ever see a 40 percent response rate like this – and these are patients who have failed to respond to multiple other drugs.”
The drug carries a potential risk of liver damage, but panelists said doctors could monitor patients for any signs. More common side effects included diarrhea, vomiting, fatigue and sleep problems.
FDA regulators are due to make their decision by late June. Approval would technically limit Epidiolex to patients with hard-to-treat forms of epilepsy. But doctors would have the option to prescribe it for other uses and it could spur new pharmaceutical research and interest into other cannabis-based products.