- A drug called Epidiolex made history last year when it became the first federally-approved prescription drug whose active ingredient comes from the marijuana plant.
- Made with ultra-high doses of the cannabis compound CBD, Epidiolex treats two rare forms of epilepsy.
- That drug may have blazed a trail that other pharmaceutical companies can follow.
- The CEO of the company behind Epidiolex believes we’re at the beginning of a "new era" in cannabis-based medicines, he told Business Insider in an interview.
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Last summer brought new meaning to the phrase "medical marijuana."
For the first time in history, US regulators approved a prescription drug whose active ingredient comes from the cannabis plant. Called Epidiolex, the drug uses ultra-high doses of the marijuana compound CBD to treat rare forms of childhood epilepsy. (One other marijuana-containing drug called Marinol is approved for nausea, but it’s made with a synthetic form of the compound THC.)
By securing a position as the first cannabis-based, federally-approved medication, Epidiolex may have blazed a trail that other drugmakers can follow.
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Almost exactly a year after Epidiolex’s approval, we’re at the very beginning of a "new era" for marijuana-based medicines, according to Justin Gover, the CEO of drugmaker GW Pharmaceuticals, which makes Epidiolex.
Epidiolex is "the first in a wholly new class of cannabis-derived medications," Gover told Business Insider in an interview.
The journey to regulatory approval for Epidiolex was long and difficult, Gover said. But as a result of GW Pharma’s work with the Food and Drug Administration, other drugmakers who are pursuing marijuana-based treatments have a clearer framework for working with regulators and seeing their treatments considered for eventual approval.
First and foremost, companies need to provide regulators with a solid body of well-designed research, he said.
"If one applies the same rigorous standards to cannabis as they do to other drugs, they should be able to get a drug approved," he told Business Insider.
GW Pharma’s path to getting Epidiolex approved
Before giving Epidiolex the official green light, FDA regulators considered the results of several large clinical trials financed by GW Pharma and focused on whether Epidiolex improved the symptoms of hundreds of children with rare, hard-to-treat forms of epilepsy.
For a clinical trial whose results were published in 2017 in the New England Journal of Medicine, 43% of children who took Epidiolex for roughly 3 months saw their number of seizures reduced by half, compared with just 27% who saw these reductions on a placebo. Of those patients who saw their seizure rates decline, 5% stopped having seizures altogether, compared with 0% of the patients given a placebo.
For another clinical trial whose results were published a year later in the same journal, researchers also tested a lower dose of the drug and concluded that on average, seizure rates declined 42% in the higher-dose group and 37% in the lower-dose group, compared to just 17% in the group given a placebo.
Regulators reviewed those results before ultimately giving Epidiolex the green light last summer.
"We’ve shown that good science can lead to approval," Gover said.
from SAI http://bit.ly/2N1X0Fe