Marijuana-derived drug OK’d for rare epilepsy disorders
MelissaJenco, News Content Editor
The Food and Drug Administration (FDA) has approved the first marijuana-derived drug
to treat two rare forms of epilepsy.
Epidiolex oral solution from GW Pharmaceuticals plc is expected to be available in
the fall to treat Lennox-Gastaut syndrome and Dravet syndrome in patients 2 years
of age and older.
Both epilepsy disorders begin in childhood, can include multiple types of seizures
and impact children’s development. Dravet syndrome is not treatable with any other
“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,” Billy
Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for
Drug Evaluation and Research, said in a news release. “In addition to another important treatment option for Lennox-Gastaut patients,
this first-ever approval of a drug specifically for Dravet patients will provide a
significant and needed improvement in the therapeutic approach to caring for people
with this condition.”
Epidiolex contains highly purified cannabidiol, which comes from the cannabis sativa
plant but does not cause intoxication, according to the FDA. When taken with other
medications during three randomized, double-blind clinical trials, Epidiolex reduced
the frequency of seizures more effectively than a placebo.
The most common side effects included sleepiness, elevated liver enzymes, decreased
appetite, diarrhea, rash, weakness, trouble sleeping and infections. Like other epilepsy
drugs, serious risks include suicidal thoughts or attempts, depression, aggression
and panic attacks. Epidiolex also may cause liver injury, according to the FDA.
Under federal law, cannabidiol is classified as a Schedule I substance with no medical
use. However, GW Pharmaceuticals said it expects the Drug Enforcement Administration
to reschedule it within 90 days so that it can be prescribed to patients.
The Academy said in a 2015 policy statement that it opposes medical marijuana that is not FDA-approved but “strongly supports
research and development of pharmaceutical cannabinoids.”
Renee A. Shellhaas, M.D., FAAP, a member of the AAP Section on Neurology, called Epidiolex
approval an advance for children with difficult seizure disorders.
“Epidiolex was evaluated in carefully designed studies in order to meet FDA requirements,”
she said via email. “It will be a big step forward for physicians to be able to prescribe
an FDA-approved form of cannabidiol to their patients.”