After approval of first cannabis-based drug in USA, what’s next?

Analysis from HempToday, the voice of the global hemp industries.

Recent approval of GW Pharmaceutical’s cannabidiol-based drug Epidiolex in the United States not only will soon give patients needed relief, but can spur further research into cannabinoids as well as expansion of the CBD sector overall. That could set off a cycle that would result in a greater number of treatment options for patients, GW has said.

GW made cannabis history June 25, 2018 when it earned approval from the U.S. Food & Drug Administration (FDA) for Epidiolex, a drug crafted as a treatment for two forms of childhood epilepsy. It is the first cannabis-derived prescription drug to gain federal approval in the United States.

The publicly traded, UK-based company has said it is “blazing a trail for any other CBD prescription medications that may follow,” and has consistently refuted accusations it is working to monopolize the cannabinoid-based medicines market, as some critics have alleged, meanwhile muddying the waters for non-prescription CBD products.

Unfounded fears

Critics have pointed to GW’s wide gallery of patents, and the protection orphan drug status provides the company in their theories about GW’s intentions.

Furthermore, some makers of non-medicinal grade CBD products have been fearful that casting CBD in a prescription framework could negatively impact sales in the fast-growing non-prescription CBD food supplements sub-sector. That’s a fear long-time cannabis veteran and consultant Richard Rose says is unfounded.

“There are many precedents for a food being a supplement but also an approved drug. It’s not as cut and dried as some think,” Rose said, noting “FDA has no intrastate jurisdiction, so I expect little impact on the dietary supplement side” in the USA.

FDA approval puts cannabidiol in the reach of medical doctors, who can prescribe it for medical conditions and have it covered by health insurance. “Prescription CBD paid by insurance is a good thing,” Rose said.

Fitting into EIHA recommendations

Epidiolex is also currently under review by authorities in the EU, where general recommendations for CBD from trade group the European Industrial Hemp Association (EIHA) bolster the case that high-dose CBD should be considered a medicinal product, “and should be regulated as such.” The Association’s approach to CBD also would not seem to predict any negative impact on the food supplements market. EIHA has proposed a three-tier regulatory system in which CBD is divided into classes for medicinal products (high doses), over-the-counter food supplements (at ‘physiological doses’) and an unrestricted food class (at low doses).

GW’s focus has been on this high-dose medical class, and their strategy clear: Cannabinoid-based pharmaceuticals that come with a pedigree and a prescription – extensive clinical trials and federal approval, targeting specific conditions.

Addressing childhood epilepsy

Epidiolex won FDA approval as a treatment for Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome, severe forms of early onset epilepsy affecting children.

Both maladies are known for the severity of the seizures they cause, and both are characterized by their drug-resistant nature. LGS treatment is often associated with a broad regimen of several anti-epileptic drugs. Until now, there has been no FDA approved treatment for Dravet Syndrome.

Epidiolex had already obtained orphan drug status for these debilitating conditions in both Europe and the United States, having undergone advanced clinical trials. It is currently under review as a treatment for LGS and Dravet by the European Medicines Agency (EMA), and due for EU approval in early 2019.

“This approval (in the USA) is the culmination of GW’s many years of partnership with patients, their families and physicians in the epilepsy community to develop a much needed, novel medicine,” Justin Gover, GW’s CEO, said in the company’s official press statement following the FDA ruling.

“These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care,” Gover said.

As part of the further approval process, Epidiolex must be rescheduled from its current Schedule I to Schedule 2 or 3 before it can be made available to patients. With the rescheduling expected to occur within 90 days. GW said it expects the drug to be available to patients by autumn 2018.

Potential for ‘off-label’ uses

Phase 3 studies of Epidiolex that were published in the prominent medical journals Lancet and The New England Journal of Medicine, found that the drug, when added to other anti-epileptic therapies, significantly reduced the frequency of the seizures associated with Dravet and LGS.

Though approved specifically for for Dravet and LGS, doctors also would be free to prescribe Epidiolex for “off-label” uses to address other conditions for which CBD may show anecdotal benefits.

FDA approval follows a recommendation earlier this year in a report from the Agency’s Peripheral and Central Nervous System Drugs Advisory Committee – a recommendation that was unanimously endorsed by committee members.

“Overall, the findings from the three Phase 3 studies demonstrate that, when added to a current anti-epileptic drug therapy, CBD oral solution reduces seizure frequency in patients with drug-resistant LGS or Dravet Syndrome while maintaining a predictable and manageable safety profile,” the April report noted.

Other CBD extracts are already widely available in the U.S., taking advantage of the 2014 Agricultural Act that effectively made hemp-derived CBD legal (although barring sales of CBD derived from marijuana).

That’s led to a burgeoning sector with estimated sales of more than $350 million in 2017 alone, and with a projected rise to more than $1.1 billion by 2022, according to some industry figures.

What’s next for GW?

Although Epidiolex was the company’s “lead candidate” for FDA approval, clinical trials and federal approval efforts are also continuing for other cannabinoid based pharmaceuticals from GW, including Sativex, a CBD mouth spray that alleviates some symptoms of multiple sclerosis, and heretofore the company’s flagship medicine.

GW also is looking at other single cannabinoid formulations, including a Cannabivarin-based pharmaceutical investigated for Autism Spectrum Disorder, other formulations for a particularly malignant form of brain tumors called glioblastoma, and even schizophrenia.

The company expects handsome returns for their efforts too, although cost-to-patient on Epidiolex has not been announced yet. Some analysts predict that Epidiolex could cost patients without health insurance between $30,000 and $60,000 per year.

Started in 1998, UK-based GW Pharmaceuticals has been active in cannabis research and the development of cannabinoid-based pharmaceuticals. The company has been researching cannabidiol since 2007.

Epidiolex will be marketed in the U.S. by Greenwich Biosciences, GW’s U.S. subsidiary. – Arne Verhoef