NDA filing by GW Pharmaceuticals for Epidiolex accepted by USFDA

LONDON: GW Pharmaceuticals plc announced that the U.S. Food and Drug Administration (FDA) has accepted for filing with Priority Review its recently submitted New Drug Application (NDA) for Epidiolex^® (cannabidiol or CBD), an investigational treatment for seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, two rare and difficult to treat conditions of childhood-onset epilepsy.

The PDUFA (Prescription Drug User Fee Act) goal date for completion of the FDA review of the Epidiolex NDA is June 27, 2018.

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 NDA accelerates the timing of the FDA review of the application compared to a standard review.

In its acceptance letter, the FDA has stated that it is currently planning to hold an advisory committee meeting to discuss this application.

“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.

“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.”

About Lennox-Gastaut Syndrome

The onset of LGS typically occurs between ages of 3 to 5 years and can be caused by a number of conditions, including brain malformations, severe head injuries, central nervous system infections, and genetic neuro-degenerative or metabolic conditions.

In up to 30 percent of patients, no cause can be found. Patients with LGS commonly have multiple seizure types including drop and convulsive seizures, which frequently lead to falls and injuries, and non-convulsive seizures.

Resistance to anti-epileptic drugs (AEDs) is common in patients with LGS. Most children with LGS experience some degree of intellectual impairment, as well as developmental delays and aberrant behaviors.

About Dravet Syndrome

Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic encephalopathy frequently associated with genetic mutations in the SCN1A sodium channels. The onset of Dravet syndrome occurs typically during the first year of life in previously healthy and developmentally normal infants.

Initial seizures are often body temperature related, severe, and long-lasting. Over time, patients with the Dravet syndrome often develop multiple types of seizures, including tonic-clonic, myoclonic, and atypical absences and are prone to bouts of prolonged seizures including status epilepticus, which can be life-threatening.

Risk of premature death including SUDEP (sudden unexpected death in epilepsy) is elevated in patients with the Dravet syndrome. Additionally, the majority will develop moderate to severe intellectual and development disabilities and require lifelong supervision and care.

There are currently no FDA-approved treatments and nearly all patients continue to experience seizures and other medical needs throughout their lifetime.

About Epidiolex^® (cannabidiol)

Epidiolex, GW's lead cannabinoid product candidate is a pharmaceutical formulation of purified cannabidiol (CBD), which is in development for the treatment of several rare childhood-onset epilepsy disorders.

GW has submitted a New Drug Application with the FDA for Epidiolex as an adjunctive treatment for seizures associated with LGS and Dravet syndrome with an expected approval and launch in 2018.

To date, GW has received Orphan Drug Designation from the FDA for Epidiolex for the treatment of Dravet syndrome, LGS, TSC and IS. Additionally, GW has received Fast Track Designation from the FDA for the treatment of Dravet syndrome and conditional grant of rare pediatric disease designation by FDA.

The Company has also received Orphan Designation from the European Medicines Agency, or EMA, for Epidiolex for the treatment of LGS, Dravet syndrome, West syndrome, and TSC.

GW is currently evaluating additional clinical development programs in other orphan seizure disorders including Phase 3 trials in Tuberous Sclerosis Complex and Infantile Spasms.