This epilepsy medication’s approval could simply be an open the door to the development of other cannabis-derived treatments.
The U.S. Food and Drug Administration (FDA) yesterday approved the first drug derived from marijuana. Epidiolex will be used to treat two rare and severe forms of epilepsy in children and young adults, Lennox-Gastaut syndrome and Dravet syndrome.
Epidiolex is made from purified cannabidiol (CBD), an ingredient found in marijuana. The medication does not contain THC, the primary psychoactive component in marijuana that causes the “high” associated with its use.
According to the FDA, Epidiolex was shown to be effective in reducing the frequency of seizures in patients with Dravet or Lennox-Gastaut syndromes. The approval was based on three clinical trials involving 516 patients with either syndrome.
Treating ‘Difficult-to-Control Seizures’
Shaun Hussain, MD, a pediatric neurologist at Ronald Reagan UCLA Medical Center in Santa Monica, says that while the FDA’s approval of a marijuana-based drug is “indeed a big deal,” it is also significant because “it offers another drug for Lennox-Gastaut and the first-ever drug for Dravet.”
“The difficult-to-control seizures that patients with Dravet syndrome and Lennox-Gastaut syndrome experience have a profound impact on these patients’ quality of life,” said Billy Dunn, MD, the director of the FDA’s division of neurology products, in a statement.
The most common side effects of Epidiolex include diarrhea, rash, insomnia, and infections. “But for the conditions the drug is treating, patients, parents, and clinicians found the side effects to be acceptable because it lowered the frequency of seizures, says Shlomo Shinnar, MD, PhD, a professor of neurology at the Albert Einstein College of Medicine in New York City and the president of the American Epilepsy Society.
Marijuana has long been known to be effective in reducing the frequency of seizures in some patients; and many patients buy medical grade marijuana in states where it is legal as treatment. “This approval, however, means patients can be assured of getting a consistent dose of the drug because of the standards set by the FDA,” says Dr. Hussain.
Issues of Classification and Cost
Marijuana is currently classified by the Drug Enforcement Agency (DEA) as a Schedule I substance, meaning it has high potential for abuse. In order for the UK-based GW Pharmaceuticals, the manufacturer of Epidiolex, to sell the drug in the United States, the DEA will have to reclassify marijuana. The FDA says it expects that to happen within the next three months.
The company has not announced pricing for the drug, but Wall Street analysts previously predicted it could cost as much as $25,000 per year. Insurers have not yet said if they will cover the cost of the drug.
Concerns and Commitment
In a call with reporters yesterday, the FDA’s commissioner of food and drugs, Scott Gottlieb, MD, said “We remain concerned about the proliferation and illegal marketing of unapproved CBD-containing products with unproven medical claims.”
But Dr. Gottlieb also held out the possibility of more approvals for marijuana-based drugs down the road.
“Today’s approval demonstrates our commitment to the scientific process and working with product developers to bring marijuana-based products to market,” he said. “Such a process ensures that any new therapies from marijuana and its constituents are safe, effective, and manufactured to a high and consistent quality.”
Dr. Shinnar believes the drug was carefully chosen, in light of the severe conditions it can help treat. “The conditions it is approved to treat are devastating and difficult to control and so rightly got the attention of pharmaceutical companies and regulatory agencies,” he says.