On June 25—and for the first time ever—the FDA an- nounced it had approved a drug containing an active ingredient found in cannabis. Cannabidiol, or CBD, is
a compound that affects users differently than the commonly
thought of “high” that comes from marijuana’s tetrahydrocan-nabinol (THC) component. Epidiolex is a strawberry-flavored,
pharmaceutical-grade, purified CBD syrup used to treat patients
suffering from Dravet syndrome and Lennox-Gastaut syndrome
(LGS), both traditionally difficult to treat forms of epilepsy.
A product of U.K.-based GW Pharmaceuticals and its U.S.
subsidiary, Greenwich Biosciences, Epidiolex—currently a
Schedule 1 substance in accordance with the parent plant of
its key ingredient—appears to bring hope to families who have
long been seeking treatment options in accordance with these
rare, severe, early-onset conditions. But just what is Epidiolex, what do we know about the conditions it treats, what
happened in clinical trials to warrant FDA approval and what
does such approval mean for the overall state of the medical
cannabis market? Laboratory Equipment talked with Stephen
Schultz, Vice President, Investor Relations, GW Pharmaceuticals, to learn more.
Epidiolex and clinical trials
The Epidiolex development program began with a single
patient whose parents had read some anecdotal evidence of CBD
artisanal products helping reduce convulsive seizures in patients.
That expanded to over 1,000 who currently receive medication
at no charge through the FDA’s Compassionate Use Access, a
program designed to assist patients who are out of options. The
program has been in place since 2013 and runs in parallel to pivotal studies that inform the new drug application for the safety
and efficacy of the drug in question—in this case, Epidiolex.
How does Epidiolex work? “We believe it’s multi-faceted,”
Schultz said. “Perhaps one of those mechanisms has to do with
inflammation management and perhaps one has to do with calci-
Still, Schultz said, it’s clear Epidiolex works on a systemic
level—something unique to cannabidiol medications, and some-
thing well-suited to treat epileptic conditions, particularly those
in which the cause of the seizures are unknown. In such in-
stances, a drug that affects the human system as a whole (rather
than having a target point in the body) can have a significant
impact on the quality of life of those living with these condi-
tions because they may be beneficially affected by the systemic
modulations. That’s important because more than 90 percent of
patients with Dravet syndrome or LGS suffer from at least one
seizure per day.
Dravet syndrome is a type of epileptic encephalopathy that is
associated with SCN1A sodium channel mutations. It affects over
5,400 people under 20 years of age in the U.S. alone. As the condition progresses, patients may suffer from multiple types of seizures—
many of which, such as status epilepticus, can be long lasting and life
threatening. Patients with this condition can suffer from behavioral
disturbances, developmental delays, intellectual disability and more.
Until the approval for Epidiolex, there were no FDA-approved
medications in the U.S. for treating Dravet syndrome. As such, the
drug—which received the formal orphan designation—was fast-tracked to allow for an abridged review period. In Phase 2 trials,
34 patients from the U.S. and U.K. were randomized and received
the drug—or a placebo—at various dosages over a course of four
weeks. After a 10-day taper, they followed up with four additional
weeks of treatment. Results published in the New England Journal
of Medicine showed a dose of 20/mg/kg (coupled with standard
treatment for epilepsy) decreased patient seizures by 39 percent—a
26 percent increase over the placebo group. The results were
further explored during a similarly structured 120-person Phase 3
trial. In this trial, 43 percent of patients experienced a more than
50 percent reduction in convulsive seizures, and almost twice as
many caregivers said the patients’ condition improved while taking
Epidiolex (compared to the placebo). Adverse effects included
diarrhea, decreased appetite, vomiting, pyrexia and others, but the
treatment was generally well tolerated.
LGS can be caused by severe head injuries, brain malformations, nervous system infections and other issues—although in
30 percent of patients, there is no known cause—and it generally
initially affects children aged 3 to 5. LGS patients often suffer
drop seizures—defined by researchers as “atonic, tonic or ton-ic-clonic seizures involving the entire body, trunk or head that led
New Cannabis Drug Granted FDA
Approval to Treat Specific
Forms of Epilepsy
What you need to know about Epidiolex.
by Jessica Burdg, Contributing Science Writer