The U.S Food and Drug Administration on 25th June approved the use of country’s first marijuana based drug. It is a medication that treats Dravet syndrome and Lennox-Gastaut syndrome (LGS), two rare epilepsy disorders that primarily affect children.
The oral liquid based drug ‘Epidiolex‘ is formulated from the cannabidiol (CBD) portion of cannabis. The use of CBD is great since it does not cause intoxication or ‘high’ in users. This is especially important for children, as it is the tetrahydrocannabinol (THC) component of the weed which has negative implication of being addictive and affects the developing brain. The recent isolation of CBD from THC has opened the doors for therapeutic use of the weed. For a really long time Drug Enforcement Administration (DEA) had placed all marijuana-based derivatives in ‘Schedule 1 Class’ and listed them as a substances of high abuse potential. This puts it out of reach for any research and implies that it has no current medicinal benefit.
Epidiolex’s approval also marks the first time the FDA has approved a drug to treat Dravet syndrome. However the appreciation didn’t come easy. The drug was approved by the FDA only after review of four large studies that looked at Epidiolex for the treatment of epilepsy disorder in both adults and children (aged two and older). After 14 weeks, the average seizure frequency was found to have dropped in all studies. In the trial the only main side effect reported was liver toxicity, which appeared in the form increased liver enzyme production. Since CBD products cannot have unreliable concentrations or poor quality the medication will need to go through several more steps before it’s available as a prescription. “Controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s drug approval process, is the most appropriate way to bring marijuana derived treatments to patients,” FDA Commissioner Scott Gottlieb, said. Thus it will take at least 90 days before the medication hits pharmacy shelves.
The London-based company behind the drug, GW Pharmaceuticals plc and Dr. Anup Patel, a paediatric neurologist at Nationwide Children’s Hospital in Ohio who is one of the lead authors of the study, is expecting another momentous decision- the US DEA to reclassify CBD. This decision would definitely easy use of marijuana based curative research.
GW CEO Justin Gover said “We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high-quality products. But, at the same time, we are prepared to take action when we see the illegal marketing of CBD containing products with serious, unproven medical claims. Marketing unapproved products, with uncertain dosages and formulations, can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases“.
Today’s approval “serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies. And the FDA is committed to this kind of careful scientific research and drug development,” FDA Commissioner Gottlieb concluded.
Overall, the approval is great news for patients suffering from Dravet syndrome or LGS.