http://www.raredr.com/news/gw-pharma-completes-nda
GW Pharma Completes NDA Submission for Cannabidiol Drug to Treat LGS and Dravet Syndrome

James Radke

Today, GW Pharmaceuticals, plc. and Greenwich Biosciences announced the completion of the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Epidiolex (cannabidiol) to help manage seizures associated with 2 rare diseases: Lennox-Gastaut syndrome (LGS) and Dravet syndrome.

LGS is a severe childhood epileptic encephalopathy associated with multiple seizure types, including tonic, atonic, tonic clonic, myoclonic, and absence seizures.

Two phase 3 clinical trials have been conducted testing Epidiolex in LGS patients. In the first, 171 patients were randomized to receive Epidiolex 20mg/kg/day (n=86) or a placebo (n=85), as well as their current antiepileptic therapy. The study found that Epidiolex reduced the median number of monthly seizures by 44% compared 22% in patients receiving placebo.
 
The difference between Epidiolex and placebo became apparent during the first month of treatment in the trial and was sustained throughout the entire treatment period.
 
Results of the second phase 3 trial were presented earlier this year at the 2017 Annual American Academy of Neurology (AAN) meeting in Boston and showed that LGS patients who are given Epidiolex had a 50% decrease in monthly seizures compared to a 15% decrease in patients on a placebo. 

Dravet syndrome is a complex childhood epilepsy encephalopathy disorder, and is often-associated with drug-resistant seizures and a high mortality rate. It almost always begins in the first year of life in an otherwise healthy infant. 

In May 2017, data from the phase 3 study testing Epidiolox in Dravet syndrome was published in the New England Journal of Medicine showing the drug to be safe and effective.

In the double-blind, placebo-controlled trial, 120 children and young adults with Dravet syndrome and drug-resistant seizures were randomly assigned either Epidiolox (20 mg per kilogram of body weight daily) or placebo, in addition to their standard antiepileptic treatment.

In the cannabidiol-treated group, the median frequency of convulsive seizures per month decreased from 12.4 to 5.9 seizures In contrast, patients receiving placebo only saw a decrease of 14.9 to 14.1 seizures per month.

Adverse events (AEs) that occurred most frequently in the Epidiolox group included diarrhea, vomiting, fatigue, pyrexia, somnolence, and abnormal results on liver-function tests. 

Per GW Pharmaceuticals: "The submission of the Epidiolex NDA is an important milestone for patients with LGS and Dravet syndrome in that a potential new treatment option is within sight for these very difficult to treat and devastating conditions," stated Justin Gover, CEO of GW Pharmaceuticals. “On behalf of all those patients, clinicians and GW employees who have supported the Epidiolex program, we are pleased to submit this promising therapy for FDA review and we look forward to working with the FDA throughout the review process. This regulatory submission is a demonstration of GW’s commitment to developing innovative cannabinoid-based treatments that have the potential to address significant unmet medical needs.”

The company plans to file a Marketing Authorization Application (MAA) in Europe for Epidiolex in the near future. A Prescription Drug User Fee Act (PDUFA) data has not yet been established.

Image courtesy wikimedia commons. 

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