British drug maker GW Pharmaceuticals released the first results on the efficacy and safety of Epidiolex, a purified form of the marijuana compound cannabidiol (CBD), on Tuesday.
The drug led to an average reduction in seizure frequency of 50% among a group of 27 children and young adults with treatment-resistant epilepsy.
“Some children have had marked reductions in their seizures”15% of patients were completely seizure-free following treatment. Of the 9 patients who suffered from Dravet syndrome, 3 that received Epidiolex became seizure-free.
“The initial open-label study with Epidiolex has provided encouraging results,” said Orrin Devinsky, MD, director at the NYU Comprehensive Epilepsy Center and one of the drug’s lead investigators.
“Some children have had marked reductions in their seizures and overall, the medication has been well tolerated.”
GW Pharmaceuticals called the results “promising” and announced plans to begin Phase 2/3 clinical trials during the second half of 2014.
So far, the trials have been conducted as open-label studies without a placebo group, which lacks the strength required for FDA approval. However, the FDA has granted Epidiolex both orphan drug status and fast track designation in an effort to speed the testing process.
All 27 patients were treated at New York University Langone Medical Center (NYU) or the University of California at San Francisco (UCSF) and underwent at least 12 weeks of treatment.
The company also released safety data gathered from a group of 62 patients who were treated for a combined total of 120 months.
The most commonly reported side effects were somnolence (40% of patients) and fatigue (26%), followed by diarrhea (16%), increased appetite (11%) and decreased appetite (10%).
So far, no serious adverse effect has been linked to the drug.