The Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for Epidiolex® (cannabidiol; Greenwich Biosciences) for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients 1 year of age and older.
Epidiolex is a pharmaceutical formulation of highly purified cannabidiol (CBD). The precise mechanism by which cannabidiol exerts its anticonvulsant effects is unknown; it does not appear to exert its effects through interaction with cannabinoid receptors.
The sNDA was approved based on data from a double-blind, placebo-controlled phase 3 trial of Epidiolex in 224 patients aged 1 to 65 years with drug-resistant epilepsy associated with TSC. Patients were randomized to receive either Epidiolex 25mg/kg/day or 50mg/kg/day, or matched placebo for 16 weeks (4-week titration and 12-week maintenance phase).
Results showed that treatment with Epidiolex 25mg/kg/day was associated with a statistically significantly greater reduction from baseline in TSC-associated seizures over 16 weeks (primary end point) compared with placebo (percentage change from baseline during treatment in estimated mean seizure frequency: -48% vs -24%, respectively; P <.01).
The most common adverse reactions (≥10% greater than placebo) reported in patients with TSC treated with Epidiolex included diarrhea, transaminase elevations, decreased appetite, somnolence, pyrexia, and vomiting.
Commenting on the approval, Douglas Throckmorton, MD, deputy center director for regulatory programs in the FDA’s Center for Drug Evaluation and Research, said: “The Agency is committed to supporting rigorous scientific research on the potential medical uses of cannabis-derived products and working with product developers who are interested in bringing patients safe and effective, high quality products.”
Additionally, the FDA has expanded the age range for all Epidiolex indications to include patients aged ≥1 year. Epidiolex is also indicated for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.
Epidiolex is supplied as a 100mg/mL strawberry-flavored oral solution. Any unused portion of the medication should be discarded after 12 weeks of first opening the bottle.
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