FDA Accepts Supplemental NDA for Cannabidiol to Treat Seizures of Tuberous Sclerosis Complex

The US Food and Drug Administration (FDA) has accepted for filing, with priority review, a recently submitted supplemental new drug application (sNDA) for the use of cannabidiol (Epidiolex, GW Pharmaceuticals, Carlsbad, CA) CV to treat seizures associated with tuberous sclerosis complex (TSC). The Prescription Drug User Fee Act (PDUFA) goal date for completion of the FDA review of the cannabidiol sNDA is July 31, 2020. Cannabidiol is already approved for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS), 2 rare and difficult to treat childhood-onset epilepsies.

“We are pleased with the FDA’s acceptance of our Epidiolex sNDA filing with Priority Review, an action that underscores the unmet need for new treatment options for patients with TSC, a rare and severe childhood-onset disease,” said Justin Gover, GW’s chief executive officer. “More than 60% of individuals with TSC do not achieve seizure control with standard antiepileptic drug treatments. Today’s news is therefore important for TSC patients and their clinicians, and we look forward to working with the FDA during the review process to expand the Epidiolex product label for use in TSC.”

Priority review status is designated for drugs that may offer major advances in treatment or provide a treatment where no adequate therapies exist. The granting of priority review for the Epidiolex sNDA accelerates the timing of the FDA review of the application to a 6-month period compared to a standard review of 10 months.

Cannabidiol a pharmaceutical formulation of highly purified cannabidiol (CBD), is the first in a new class of antiepileptic medications with a novel mechanism of action, and the first prescription plant-derived cannabis-based medicine approved by the FDA. In the US, cannabidiol is indicated for the treatment of seizures associated with LGS or DS in individuals age 2 years or more. A sNDA has been submitted to the FDA for the treatment of seizures associated with TSC. Cannabidiol has received approval in the European Union under the tradename cannabidiol for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and DS. Cannabidiol has received orphan drug designation from the FDA and the EMA for the treatment of seizures associated with DS, LGS and TSC, each of which are severe childhood-onset, drug-resistant syndromes.