Cannabis drug sparks bright hope for children with severe epilepsy as it showed promise in clinical trials.
GW Pharmaceuticals, a biopharmaceutical firm that aims to discover and develop drugs from cannabis, announced on Monday the positive results of the first pivotal Phase 3 study of its experimental drug, Epidiolex.
The drug was able to reach the main target of eliciting more significant decrease in convulsive seizures than placebo in the span of the study period.
"The positive outcome of this Phase 3 trial is a significant milestone in the development of Epidiolex as a potential new treatment for patients suffering with Dravet syndrome," says GW's CEO Justin Gover.
Dravet Syndrome is a rare and severe type of epilepsy that has no existing FDA-approved treatment yet. For this, Gover says the company is excited about the possibility that Epidiolex will be the first.
The Clinical Trial
The study involved 120 participants who were randomly assigned to receive either Epidiolex or placebo, in addition to their current anti-epileptic treatments.
The average age of the participants was 10 years old and 30 percent were less than 6 years old. The median convulsive seizure frequency per month is 13.
The scientists compared the percentage change in monthly seizure episodes during the four-week baseline observation and the 14-week treatment periods.
The findings show that the Epidiolex group had a median decrease of 39 percent in monthly convulsive seizures. The placebo group showed a 13 percent reduction. Such difference is considered statistically significant; thus, achieving the set endpoint of the clinical trial.
The participants generally tolerated Epidiolex during the study. Among the most common adverse effects noted were diarrhea, decreased appetite, vomiting, fatigue, increased sleepiness, fever, upper respiratory tract infection and convulsion.
The severity of these adverse effects were mild to moderate in 84 percent of the patients who experienced it.
Serious adverse effects were noted in 10 patients in the Epidiolex group, compared to only three in the placebo. Eight Epidiolex patients and one placebo patient halted treatment due to adverse reactions.
GW says it will release additional information through its publications and medical conferences.
What The Future Holds
Gover says the company now plans to request a meeting with the FDA to talk about the proposed regulatory submission for the drug.
GW is also looking forward to the results of the second pivotal trial for Dravet syndrome and two Phase 3 trials for Lennox-Gastaut syndrome, which is another type of epilepsy.