For the first time, this means that patients will be able to receive a treatment with a primary ingredient, purified cannabidiol (CBD), that is derived from the cannabis plant. The US Food and Drug Administration (FDA) approval was based on three Phase III trials, and an open-label extension study, looking into the safety and efficacy of the treatment.
Despite these studies showing that the treatment reduced the number of seizures in patients, when compared with placebo, there had still been no precedent to date to indicate how the FDA would react to the application.
in-PharmaTechnologist reached out to GW to learn more about the process of working with the FDA to navigate the approval process and a spokesperson replied detailing the ways in which the Agency had moved to speed up the process: “The FDA granted priority review designation for the application. Fast-track designation was granted for Dravet Syndrome and Orphan Drug designation was granted for both the Dravet Syndrome and Lennox-Gastaut Syndrome (LGS) indications.”
They added, “In April 2018, an independent advisory committee to the FDA voted unanimously in favour of approving the medicine.”
The last point refers to the 13-0 vote that carried through the advisory committee to approve the drug, which indicated, at the time, that an approval looked likely.
However, there is now one further hurdle that GW’s product will need to surmount to get its product onto market and which leaves the final decision on the drug down to the Drug Enforcement Administration (DEA).
As currently cannabis is a Schedule I substance, it is considered to have no medical benefits. This means that the DEA will have to evaluate, within 90 days, whether it is prepared to change the definition for Epidiolex to make it available to patients.
This is not stopping GW from making future plans for the drug’s use, as a spokesperson for the company revealed: “GW will continue to focus its efforts on educating physicians that treat patients with LGS and Dravet Syndrome about the safety and efficacy of Epidiolex in these specific populations. Meanwhile, the company will continue to study CBD for the treatment of other difficult-to-treat diseases like tuberous sclerosis complex, and work with the FDA to obtain approval for additional indications if the data warrant.”
When asked about how working with the FDA will evolve into the future, the spokesperson referred to the statement put out by FDA Commissioner, Scott Gottlieb, which stressed that high-quality research in the area will be expected for medicinal claims of cannabis-derived products to be reviewed.
In the statement, the Commissioner said: “This product approval demonstrates that advancing sound scientific research to investigate ingredients derived from marijuana can lead to important therapies. This new treatment provides new options for patients. This is an important medical advance. But it’s also important to note that this is not an approval of marijuana or all of its components.”
With GW’s product projected by Clarivate Analytics to reap sales of $1.2bn (€1.03bn), there is likely to be a surge of interest in researching and producing the ingredients necessary to create potential new treatments.
Gottlieb’s comments can then be seen as an attempt to temper inevitable excitement in the area by stressing the need for serious science behind any application to the Agency.
It is clear that the area is a rapidly growing field and, just last week, in-PharmaTechnologist spoke to Axim Biotechnologies about the FDA granting it a patent on extraction process of Delta-9-tetrahydrocannabinol for its use as a potential drug candidate.