Questions abound about availability, legality of new cannabis-derived epilepsy drug


The first-ever federal approval of a marijuana-derived drug, Epidiolex (cannabidiol), comes with a cloud of complications.

Dr. Jacqueline A. French, professor of neurology at New York University and chief scientific officer of the Epilepsy Foundation

Dr. Jacqueline A. French

The drug, approved by the Food and Drug Administration in June to treat Lennox-Gastaut syndrome and Dravet syndrome in patients aged 2 years or older, is expected to be so expensive that those seeking it for off-label uses may join those who buy unregulated cannabis products on the open market. And legal complications could prevent some neurologists from prescribing Epidiolex at its planned fall launch.

Epidiolex is an oral solution of purified cannabidiol (CBD), a component of the marijuana plant that does not make people high.

According to the results of a phase 3 trial in patients with Lennox-Gastaut syndrome published earlier this year, median monthly drop seizures fell by 43.9% in a Epidiolex group compared with 21.8% in a placebo group (Lancet. 2018;391[10125]:1085-96). A 2017 study found that patients with Dravet syndrome who took the drug had fewer median convulsive seizures per month, dropping from 12.4 to 5.9, while there was barely a difference for the placebo group (N Engl J Med. 2017;376:2011-20). In both trials, patients received Epidiolex as an add-on treatment.

“There definitely are side effects such as sleepiness, abnormal liver function values, and other things that people will have to watch out for, such as interactions with other drugs,” Jacqueline A. French, MD, professor of neurology at New York University and chief scientific officer of the Epilepsy Foundation, said in an interview. “But it will definitely be a great benefit to some and a benefit to many.”

It’s not clear how the drug works, she said.

Greenwich Biosciences, the U.S. subsidiary of British drug maker GW Pharmaceuticals, plans to market Epidiolex this fall, spokesman Steve Schultz said in an interview. First, however, the U.S. Drug Enforcement Agency must reschedule the drug so it doesn’t fall under federal antimarijuana laws. It has 90 days after the FDA approval to do so, and its rescheduling is expected.

Then the focus will turn to the states, which have a crazy quilt of laws about marijuana and its medical use. “We’ve worked with the state legislators to enact laws or to modify laws to allow for our medicine to be made available,” Mr. Schultz said. “There may be one or two that we’re still working on.”

At issue: States and the District of Columbia have a wide variety of laws that appear to affect the sale of Epidiolex.

According to the website procon.org, 30 states and the District of Columbia allow medical marijuana, although the laws vary regarding what is allowed and do not address cannabis-derived pharmaceuticals.

Another 17 states have laws that address CBD specifically, often with language that allows its use to treat epilepsy in all or specific cases, according to procon.org.

Rod Kight, a lawyer based in North Carolina

Rod Kight

The Kentucky law, for instance, only allows prescriptions by hospitals and clinics linked to public universities. A law in Missouri, according to procon.org, only allows the use of certain kinds of “cannabis oil” for intractable epilepsy and “requires a neurologist to determine that the patient did not respond to at least three treatment options to be eligible to use the marijuana extract.”


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