FDA Accepts New Drug Application for Lennox-Gastaut and Dravet Syndromes Treatment
This morning, GW Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s new drug application (NDA) for Epidolex (cannabidiol) as an adjunctive treatment for seizures in Lennox-Gastaut syndrome (LGS) and Dravet syndrome, a pair of rare, severe childhood-onset epilepsy disorders.
The NDA was submitted at the end of October
. The Prescription Drug User Fee Act (PDUFA) goal date for completion of the FDA review of the Epidiolex NDA is June 27, 2018.
GW Pharmaceuticals had previously received Rare Pediatric Disease and Orphan Drug Designations from the FDA for Epidiolex for both patient populations, and was previously granted Fast Track Designation for the therapy in Dravet syndrome.
LGS is a severe epileptic encephalopathy characterized by multiple seizure types, including: tonic, atonic, tonic clonic, myoclonic, and absence seizures. Dravet syndrome is complex, and often-associated with drug-resistant seizures and a high mortality rate.
“We are pleased with the FDA’s acceptance of our NDA filing with Priority Review, an action that underscores the unmet need in the LGS and Dravet syndrome populations,” said Justin Gover, GW’s Chief Executive Officer in a press release. “We look forward to working with the FDA during the review process to support the case for approval of Epidiolex so as to provide a much-needed new treatment option for patients that suffer from these highly treatment-resistant conditions of childhood-onset epilepsy.”
Epidiolex is GW Pharmaceuticals’ lead cannabinoid product candidate. It is a pharmaceutical formulation of purified cannabidiol (CBD), which is in development for the treatment of several rare childhood-onset epilepsy disorders. In Phase 3 studies, it has exhibited the ability to reduce the median number of monthly seizures by double what placebo is capable of in patients with LGS. Additionally, in a Phase 3 study of patients with Dravet syndrome, it reduced the median frequency of convulsive seizures per month from 12.4 to 5.9. Comparatively, patients administered placebo only saw a decrease from 14.9 to 14.1 seizures monthly.
In its acceptance letter, the FDA has stated that it is currently intending to host an advisory committee meeting to discuss the application.
GW Pharmaceuticals has also received Orphan Designation from the European Medicines Agency (EMA) for Epidiolex for the treatment of LGS and Dravet syndrome, as well as West syndrome and tuberous sclerosis complex (TSC). Additional clinical development programs in other orphan seizure disorders, including Phase 3 trials in Tuberous Sclerosis Complex and Infantile Spasms, are also being evaluated.
For more from the FDA, including applications, designations and approvals, follow Rare Disease Report