An experimental cannabis drug, Epidiolex, has been successful in treating children with a form of childhood epilepsy according to the results of a much-awaited clinical trial. This has gone on to double the value of shares of its makers GW Pharmaceuticals, much to the cheer of investors. Experts and drug analysts opined that this could propel the experimental drug’s sales to over $1 billion.
Cannabis Drug – Success in Trials
UK’s GW Pharmaceuticals is nearly on its way to getting FDA’s approval for a cannabis drug Epidiolex which passed the phase 3 clinical trial to prove its ability to treat children with a rare form of epilepsy. About 120 children with Dravet Syndrome, a congenital disorder, took part in the trial. This is the first trial out of the six that are planned or ongoing. The children were aged about 10 years.
The patients were also those that experienced an average frequency of 13 seizures in a month when the study started. Half of the recruits were randomly assigned to receive the cannabis drug Epidiolex, whereas the others received a placebo. At the end of the study period, the group that took Epidiolex had a median 39% reduction in the number of seizures experienced in a month’s time. The group that received the placebo had only a 13% reduction in the seizure numbers. According to the researchers, this result has high significance statistically.
Currently, there are two prescription cannabis drugs based on THC, the main active ingredient in cannabis. However, Epidiolex is the first ever drug that has been made with cannabidiol, the plant’s second main active ingredient. It is interesting to note that there is yet another drug for multiple sclerosis, a creation of GW Pharmaceuticals, which carries a combination of THC and cannabidiol. This drug is not yet available in the United States.
Dravet Syndrome is a difficult-to-treat epilepsy condition. According to Dr. Orrin Devinsky, the success of the cannabis drug in the phase 3 trial reflects the efficacy and safety of pharmaceutical cannabidiol on children with the condition.
Some of the mild side effects experienced by the children in the trials were drowsiness, diarrhea, and reduced appetite. About ten patients experienced severe side effects and 8 stopped the treatment altogether. In the placebo group, only 1 patient discontinued with the treatment. About 84 percent of the patients that experienced side effects rated them as mild or moderate.
Prospects for GW Pharmaceuticals
With positive results that came of the phase 3 clinical trial of Epidiolex, the share prices of GW Pharmaceuticals more than doubled. In intraday trading, the company’s stock went up by 139 percent. The cannabis drug is predicted to generate sales of over $1.1 billion by the end of the decade for the company.
This study of the cannabis drug is the first one of the four phase 3 trials that was slated to take place. The results are expected this year and will confirm the healing benefits of the cannabidiol, derived from marijuana. Justin Gover, CEO of GW Pharmaceuticals, said that the company is excited about the prospects of Epidiolex becoming the first ever FDA-approved drug for the treatment of Dravet Syndrome in children. In this connection, the company officials are also planning a meeting with US FDA to discuss methods of seeking regulatory approval for the cannabis drug.
Interestingly, Epidiolex, the cannabis drug that is administered as syrup to children, is being additionally tested in phase 3 trials for yet another rare form of epilepsy, Lennox Gastaut Syndrome. The results are expected this year. The drug is due to be tested for its use in tuberous sclerosis complex, another indication in epilepsy. In the meanwhile, the company has also been scaling production capability of the cannabis drug in the light of the new developments.
According to GW, as many as 30 percent of child epilepsy patients are not helped by using the epilepsy medicines that are currently available in the market. They are dependent on the marijuana extracts that are sold by medical marijuana dispensaries. Company sources also said that hundreds of patients, both children and younger adults, have been using the cannabis drug Epidiolex under specific programs (outside trials) which allow them to have the drug.