The US Food and Drug Administration (FDA) has given the thumbs up to a purified formulation of cannabidiol (CBD) (Epidiolex oral solution, GW Pharmaceuticals) for the treatment of seizures related to two rare forms of epilepsy in patients older than 2 years of age. The two types of epilepsy indicated are Lennox-Gastaut syndrome and Dravet syndrome.
"This is the first FDA-approved drug that contains a purified drug substance derived from marijuana," the agency announced in a press release. "It is also the first FDA approval of a drug for the treatment of patients with Dravet syndrome."
"This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies," FDA Commissioner Scott Gottlieb, MD, said in the same release.
"The FDA is committed to this kind of careful scientific research and drug development," added Gottlieb.
He noted that marijuana-derived therapies are most appropriately brought to patients through strict clinical trials that assess both safety and efficacy, and then by going through the FDA's stringent drug approval process.
The current drug was evaluated in three randomized, placebo-controlled studies with a total of 516 patients evaluated — all with Dravet syndrome or Lennox-Gastaut syndrome. Results showed that add-on therapy with the CBD product was more effective than placebo in reducing seizure frequency.
Because of these "well-controlled" trials, which were published in the New England Journal of Medicine and Lancet, "prescribers can have confidence in the drug's uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes," said Gottlieb.
Commonly reported adverse events in patients receiving the add-on treatment included elevated liver enzymes, sedation and lethargy, sleepiness, decreased appetite, fatigue, insomnia and poor sleep quality, infections and rash, and diarrhea.
Retrieved from: https://www.medscape.com/viewarticle/898510