For the first time, the Food and Drug Administration has approved a cannabis-based medication. It’s called Epidiolex, and it is now available via prescription in every state. The drug is an oral solution and is approved for use by patients aged 2 and up.
Epidiolex was developed to treat a rare genetic condition known as Dravet Syndrome. The disease begins in the early stages of life. It is also intended for use in the treatment of Lennox-Gastaut syndrome, which is a form of epilepsy that also begins in early childhood.
Justin Gover, the CEO of GW Pharmaceuticals; maker of Epidiolex, wrote, “Because these patients have historically not responded well to available seizure medications, there has been a dire need for new therapies that aim to reduce the frequency and impact of seizures. We are committed to ensuring that these patients can access this novel cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and is eligible to be covered by insurance for appropriate patients.”
The drug was recommended for approval by an advisory committee earlier this year and was approved by the FDA a month later. The Department of Justice classified the drug as a Schedule V controlled substance, making it legal for doctors to prescribe it to patients. CBD and marijuana are still classified as Schedule I controlled substances.
FDA Commissioner Dr. Scott Gottlieb wrote in a press release, “Adequate and well-controlled clinical studies supported Epidiolex’s approval, so prescribers can have confidence in the drug’s uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes. The FDA will continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and stand ready to work with product developers who are interested in bringing patients safe and effective, high-quality products.”
Dr. Orrin Devinsky, the director of the NYU Comprehensive Epilepsy Center was the leading investigator during clinical trials for the drug. He said, “In those syndromes, when Epidiolex was added to three other seizure drugs, on average, it reduced convulsive seizures by about 25% compared to a placebo. I think it’s very clearly effective, and was statistically significant in all three of the large studies we did. But, the effect was modest. There are some people who had dramatic improvements. Many had a modest improvement, and some had no improvement. So it’s not a miracle drug. It’s an effective drug, and I think its side effect profile is quite good compared to other seizure drugs that we have, but it’s not a miracle cure.”
The placebo effect is generally considered to have an effectiveness rate of 55 to 60% across all clinical trials. For a drug to be considered viable, it has to rise above that rate. Drugs that fail to provide a better than 60% rate of improved medical outcomes are not believed to justify any of the side effects.
The most frequent side effects of the drug are, lowered appetite, drowsiness, and increased liver enzymes.
Experts familiar with the drug rank its effectiveness between “fair” and “very effective.” The FDA’s acceptance of the drug has signaled what could lead to a general acceptance of the use of cannabis products for mainstream medical use.
GW Pharmaceuticals lists the drug’s annual cost at $32,500. They say the price is “commensurate with other branded, FDA-approved anti-epileptic drugs (AEDs), such as Banzel, and access for eligible patients is expected to be similar to such AEDs for similar indications for LGS such as Onfi and Banzel.”
While this may be a step forward for cannabis products being more broadly accepted by the medical community, stories of natural marijuana being used to treat seizure disorders are centuries old. Medical marijuana was cited in Scientific American in 2013 for its ability to treat Dravit syndrome where all other medical interventions proved disappointing.
Neurologist and pediatrics professor, Kevin Chapman told Scientific American, “I was a little surprised that the overall number of side effects was quite high but it seems like most of them were not enough that the patients had to come off the medication. I think the study provides good data to show that medical marijuana is safe- the adverse effects are mostly mild.”
Cannabis trials are still more promising than those for this new drug. This raises the question, ‘why do we need a pharmaceutical extract of marijuana when actual marijuana is more effective?’ It’s a question that we do not expect to be addressed in a forthright manner by the medical establishment.