An experimental marijuana-based drug for treating severe childhood epilepsy won a favorable review from U.S. Food and Drug Administration on April 17, signaling hope that it could come to the market soon.
The UK's GW Pharmaceuticals, the maker of the drug, provided the FDA "substantial evidence" of the medicine's effectiveness, FDA staff confirmed in a report. The drug is supposed to treat seizures associated with two rare forms of childhood epilepsy. The medication appears to yield increased risks of developing a liver injury, though it's manageable, according to the report.
"The risk-benefit profile established by the data in the application appears to support approval of cannabidiol," FDA staff said in its review, as Bloomberg reports.
Epidiolex Seeking FDA Approval
The medicine is Epidiolex, which will be taken as a strawberry-flavored syrup. It's a purified form of the aforementioned cannabidiol, an active ingredient found in marijuana. Even still, it only contains less than 0.1 percent of tetrahydrocannabinol, which is what induces "highness" among cannabis users.
Epidiolex will treat children suffering from Dravet syndrome, a severe form of epilepsy that involves myoclonus-type seizures, among others, and Lennox-Gastaut syndrome, another severe form of epilepsy distinguished by multiple types of seizures and intellectual disability. Both these are among the most resistant to medication.
Overall findings from several clinical studies illustrated that, on top of existing therapy, Epidiolex "reduces seizure frequency in patients with drug-resistant [Lennox-Gastaut syndrome] or [Dravet syndrome] while maintaining a predictable and manageable safety profile," according to FDA staff.
An advisory committee meeting is scheduled for April 19, where outside agency advisers will weigh in on the proposed medicine. Their recommendation will play a key role in the FDA's approval decision.
What This Means For Future Marijuana-Based Medicine
Epidiolex's approval could pave the way for other cannabis-derived medication, according to GW Pharmaceuticals CEO Justin Gover, suggesting that it "will mark a sea change in the acceptability of cannabinoids as therapy." If this drug gets approved, it's easy to imagine for other cannabidiol-based treatments to have great chances at getting approved as well.
The FDA is scheduled to make its final decision this June 27.
Data from the Centers for Disease Control and Prevention says 4.3 million Americans suffer from epilepsy. While the medicine in question will only be able to treat two rare types, the company plans to explore treatments for other forms as well.
What's more, GW Pharmaceuticals is also looking into manufacturing treatments for other childhood-related diseases, such as infantile spasms.