On Monday the U.S. Food and Drug Administration (FDA) approved Epidiolex, GW Pharmaceuticals’ (NASDAQ:GWPH) CBD-based drug created to treat two rare forms of childhood epilepsy. The FDA’s decision means that Epidiolex is the first-ever approved prescription drug made from cannabidiol.
Earlier this year, the FDA convened an advisory panel whose members unanimously supported the drug’s approval, citing a tremendous body of research evidence compiled by GW Pharmaceuticals in the process. The company studied the drug in over 500 epileptic children and adults.
What It Does
The drug was approved for treatment of the rare but severe epileptic disorders Dravet syndrome and Lennox-Gastaut syndrome (LGS), both of which appear at a very early age, and both of which produce multiple daily seizures known as “drop” seizures. Detrimental to children, these seizures can cause both developmental as well as intellectual disabilities and even result in death in severe cases. Indeed, both syndromes are characterized by a higher early mortality rate, and both are not typically responsive to standard epilepsy drugs. Moreover, both diseases are very rare: about 6,000 Americans have Dravet, while around 30,000 suffer from LGS.
A New England Journal of Medicine study published in May was the first large-scale clinical trial designed to study the effects of Epidiolex. In the study, patients were administered two different doses of the drug. Results showed that patients experienced fewer epileptic seizures. Patients continued to take their conventional anti-seizure medications, many of which are ineffective at controlling all the seizures, but also took either a 10mg or 20mg dose of Epidiolex. After 28 days, patients experienced a 42 percent reduction in seizures. By comparison, the placebo group experienced a 17 percent drop.
The First of Its Kind
In the past, the FDA approved synthetic cannabis drugs like Marinol, used to combat severe weight loss in cancer and HIV patients. However, Epidiolex marks the first approval of a natural, non-synthetic cannabis drug.
Parents of children with these ailments are rejoicing; many have previously taken tremendous measures to obtain Epidiolex to treat their children, some of whom suffer hundreds of uncontrollable seizures each week. The company even treated children free of charge on its compassionate access program. Before, many American families would relocate to states that legalized cannabis. Now they don’t have to do that anymore.
As an example, several years ago, Sam Vogelstein’s family approached GW for help with their 11-year-old son’s severe epilepsy. Sam was given a purified CBD product, the precursor to Epidiolex; he has been free of seizures for more than two years. Sam testified at the FDA advisory panel.
Physicians say it is of critical importance to have a well-tested, government-controlled drug like Epidiolex. FDA commissioner Scott Gottlieb remarked that the FDA is committed to careful drug development and proper evaluation of active ingredients as was done in the Epidiolex approval. The drug contains CBD specifically bred to have high concentrations for medical purposes. It contains 100 mg/ml of CBD dissolved in sesame oil, and also contains a sweetener, a strawberry flavoring agent, and ethanol.
What It Means For GW Pharmaceuticals
GW expects to have the drug on U.S. shelves in a few months, no later than fall 2018. The FDA approval will likely influence the European Medicines Agency to approve the use of Epidiolex in the European Union; if approved, European doctors could start prescribing it by summer 2019.
While GW Pharmaceuticals has not yet set a price for the drug, Wall Street analysts have estimated a cost of $25,000 per year, while forecasting that Epidiolex will likely be a blockbuster drug for the company, with annual sales that could exceed $1 billion just in the U.S. alone.
The company’s initial foray into cannabis-based medicine was its Sativex product, a sublingual spray for treating the spasticity that accompanies multiple sclerosis (MS). Sativex became the first cannabis-based medicine licensed in the UK.
The company stated it has enough medication to treat all 36,000 Americans who suffer from Dravet and LGS. They are also studying other cannabinoid-based drugs as a treatment for other diseases.
Cornering The Market
Some in the industry have accused GW Pharmaceuticals of attempting to disrupt the market and create a monopoly, though company executives say that’s not true. They stated that their drug’s FDA status is very limited, only to patients with the two rare syndromes, and as such, they cannot possibly create a monopoly. However, the FDA approval does give doctors an option to prescribe the drug for other uses.
However, the already established industrial-hemp CBD oil industry argues that their product is not only available from online retailers and specialty shops without a prescription right now, but unlike Epidiolex it is also used for a whole host of ailments besides childhood epilepsy. It’s left the company open to criticism over just how groundbreaking their drug is, considering the alternatives available.
Earlier this year, GW Pharmaceuticals tried to upend the open CBD market. According to some outlets such as Leafly and The Cannabist the company sent lobbyists to local governments across the country to try and legislate a monopoly over the CBD market.