Rare Disease Report

Cannabinoid and Infantile Spasms

JUNE 21, 2016
James Radke, PhD
GW Pharmaceuticals continues to find new indications for their cannabinoid product, Epidolex.
Today, the company announced a 4th orphan indication for Epidiolex – infantile spasms – will be studied in a pivotal Phase 3 study beginning in the 4th quarter of 2016. The FDA has granted Epidiolex with an orphan designation for infantile spasms.
Epidiolex is already being developed in 3 other orphan indications – Dravet syndrome, Lennox-Gastaut syndrome (LGS) and Tuberous Sclerosis Complex (TSC).
In March 2016, GW announced positive results from the first Phase 3 trial in Dravet syndrome. Results of the first Phase 3 trial in LGS are expected in June 2016.
In a press release, Justin Gover, CEO of GW Pharmaceuticals said, “Currently, there are limited treatment options for children suffering from infantile spasms and outcomes for patients with the disorder include higher mortality, ongoing development of additional seizure disorders as the patient matures, and often severe cognitive and developmental delay.”
With positive results with the Phase 3 Trials with patients with Dravet syndrome, GW hope to see positive results with Epidolex for patients with Infantile Spasms.
Approximately 2,000 to 4,000 new cases of infantile spasms occur in the United States each year. This is a particular type of seizure that usually occurs in the first year of life, typically between 4-8 months of age. Research shows that an average of 50-70% of patients with infantile spasms develop other seizure types later in development.
In previous research and trials, cannabidiol has reduced patient seizures for a variety of different diseases. Last year, we reported on the drug’s ability to decrease seizures by 54% in a 12 week study involving 137 Dravet syndrome and LGS patients.
 It may also be effective in managing seizure activity in TSC although those studies are in early stages of development.
GW Pharmaceuticals is being very aggressive in gaining orphan designations for a number of rare seizure indications. Let’s hope the data for all of these clinical trials provide the regulatory bodies with adequate information to approve this marijuana-based drug.

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