The cannabis component CBD moved one step closer to approval as a pharmaceutical prescription drug when the British pharma firm GW Pharmaceuticals published its third study this year showing that its CBD significantly decreased seizures on two different rare types of epilepsy conditions.
In the study, the purified, 100 percent CBD compound, called Epidiolex, was associated with a 42 percent drop in monthly seizures.
“We will use the data from our placebo-controlled trials,” said Alice Mead, vice president of U.S. professional relations at GW Pharmaceuticals, “as well as other preclinical and clinical studies, to submit a New Drug Application” to the FDA. The goal is to get CBD approved as a pharmaceutical drug.
These study results are a double-edged sword. On the one hand, they demonstrate results that align with many anecdotes coming out of Colorado that CBD can help children with seizures. On the other hand, CBD is marketed for a range of health states and is widely sold online, in marijuana dispensaries and in health food stores—and if the FDA declares that the only form of CBD that can legally be sold is the FDA-approved pharmaceutical, the agency might find itself at the center of a revolution.
Already, federal regulatory bodies are trying to stuff the marijuana genie back into the bottle, between the DEA’s August opinion to leave marijuana as a Schedule 1 controlled substance—no medical use and with a high chance for abuse—and the FDA issuing online opinions that CBD should not be legal as a dietary supplement.
However, most Americans are living in states that have legalized marijuana for medical purposes, and four states have legalized it for the fun of it. On election day 2016, five states will be voting for recreational marijuana and another four on medical marijuana.
Despite the growing acceptance of cannabis at the state level around the country, Mead said it really has no bearing on GW’s fortunes with CBD.
“The FDA will assess our NDA based only on our scientific data,” she said, “irrespective of the drug source material.”
She said the company is investigating a number of other cannabinoids and cannabinoid formulations, with the primary research areas being epilepsy, autism, glioma and neonatal hypoxic ishemic encephalopathy.
Mead also said the company “does not comment” on other CBD products or the federal government’s take on CBD or other medical marijuana components.
“Our goal is to conduct clinical research and generate safety and efficacy data on Epidiolex to support a comprehensive review by FDA and approval as a prescription medicine that will deliver consistency and quality for DS and LGS patients,” she said.
Under the FDA’s novel Investigational New Drug (IND) protocol, GW Pharma has already published studies showing reduced seizures for its Epidiolex brand CBD when used in conjunction with traditional pharma-based standard of care against Lenox-Gastaut syndrome (LGS)—a rare and severe form of childhood-onset epilepsy.
The company has another CBD-based compound, Sativex—a blend of 2.7mg CBD and 2.5mg THC—shown to be effective for multiple sclerosis spasticity.
“Sativex is approved in 28 countries, not in the U.S., for moderate to severe spasticity due to MS for patients who have not responded adequately to other treatments,” said Mead.
In the Phase 3 randomized, double-blind, placebo-controlled trial, researchers enrolled 225 children and adults with a confirmed diagnosis of drug-resistant LGS currently uncontrolled on one or more concomitant anti-epileptic drugs. Researchers divided them into three arms—placebo, 20mg/kg body weight Epidiolex and 10mg/kg body weight Epidiolex.
On average, patients were taking approximately three drugs, having previously tried and discontinued an average of seven other drugs.
During the treatment period, patients taking Epidiolex 20mg/kg/day achieved a median reduction in monthly drop seizures of 42 percent, compared with a reduction of 17 percent in patients taking placebo, and patients taking Epidiolex 10mg/kg/day achieved a median reduction in monthly drop seizures of 37 percent.
“Both dose levels were statistically significant compared to placebo,” said Mead.
For a 75kg adult (165 pounds), the 20mg/kg daily dose is equivalent to about one-half tablespoon of oral CBD solution twice a day. The CBD is dissolved in sesame oil and delivered with a needle-less syringe.
The trial follows a June 2016 trial with positive results on Epidiolex for treating seizures associated with Lennox-Gastaut sydrome.
“All three trials have been consistent in demonstrating statistically significant reductions in seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients who added Epidiolex to their existing medications over placebo," Mead said.
“We are very pleased to report this second positive phase 3 trial in seizures associated with Lennox-Gastaut syndrome,” said Justin Gover, GW’s CEO. “This is the third positive Phase 3 trial for Epidiolex reported in 2016. All three trials provide GW with robust evidence to support the efficacy and safety of Epidiolex. This latest trial also shows that Epidiolex likely has an effective dose range, allowing for dose flexibility to address individual patient needs. These compelling results make us more determined than ever to make this important new medicine available to patients who suffer from these treatment-resistant childhood-onset epilepsies.”
Following the success of the first Dravet syndrome Phase 3 trial earlier this year, GW requested a pre-New Drug Application (NDA) meeting with the FDA to discuss a proposed Dravet syndrome NDA. The company anticipates approval from the FDA in 2017 for its CBD products for both Dravet syndrome and LGS.
Epidiolex already has Orphan Drug Designation from the FDA for the treatment of LGS, Dravet syndrome, Tuberous Sclerosis Complex and Infantile Spasms.