By O’Shaughnessy’s News Service, June 17 2014

Since the start of 2014, the British company GW Pharmaceuticals has been providing Epidiolex, a Cannabis extract that is 98% CBD, to pediatric epilepsy patients being treated by neurologists at medical centers in the U.S. On June 17 the company announced efficacy and safety data on the first 27 patients to have been treated for 12 weeks by Orrin Devinsky at NYU School of Medicine and Roberta Cilio at UC San Francisco. Each patient’s frequency of seizures had been determined prior to treatment with Epidiolex to establish their baseline.

From GW’s informative announcement:

• 48% of all patients obtained at least a 50% reduction in seizure frequency compared to baseline seizure frequency. • 41% of all patients obtained at least a 70% reduction in seizure frequency. • 22% of all patients obtained at least a 90% reduction in seizures. • At the end of 12 weeks, 15% of all patients were seizure-free.

The 27 patients were predominantly children with an average age of 10.5 years (26 of the 27 patients were between three to 18 years of age; one patient was 26). In all cases, Epidiolex was added to current anti-epileptic drugs (AEDs). On average, patients were taking 2.7 other AEDs.

There were nine Dravet Syndrome patients and 18 patients with a range of other treatment-resistant epilepsies. The Dravet patients ranged from three to 16, with an average age of 8.3 years. In this group:

Mean reduction in seizure frequency was 52% and median reduction in seizure frequency was 63%.

56% of Dravet patients obtained at least a 50% reduction in seizure frequency.

44% obtained at least a 70% reduction.

33% obtained at least a 90% reduction.

At the end of 12 weeks, 33% of Dravet patients were seizure-free.

Safety data on 62 patients were made available by GW:

• At least one adverse event was reported in 81% (50) of patients • The most common adverse events were: somnolence (40%), fatigue (26%), diarrhea (16%), decreased appetite (11%), and increased appetite (10%).

80% of reported adverse events were mild or moderate.

There were no withdrawals from treatment due to adverse events.

There was one withdrawal from treatment due to lack of clinical effect. An additional two patients are currently being gradually withdrawn from treatment after three months due to lack of clinical effect.

Serious adverse events were reported in seven patients, including one death of a patient from SUDEP (sudden unexpected death in epilepsy). None of these serious adverse events, including the one reported death, were deemed related to Epidiolex by independent investigators.

In some instances, the addition of Epidiolex may be associated with changes in serum concentrations of concomitant anti-epileptic drugs.

An interview with the chairman of GW Pharmaceuticals, Geoffrey Guy, MD, will appear in the upcoming issue of O’Shaughnessy’s.