Epidiolex, made by GW Pharmaceuticals, is the first medication derived from the cannabis plant ever approved by the FDA. The drug is made of cannabidiol (CBD), a component of marijuana that doesn't give users a high.
"There's a drug route and a pharmaceutical route and there's a dietary supplement route and we need to have both of those options available", said Jackson. "This is the approval of one specific CBD medication for a specific use", Gottlieb said.
Patel helped run trials here testing Epidiolex to treat Dravet syndrome and Lennox-Gastaut syndrome, two rare, often drug-resistant forms of epilepsy that start in childhood. Others said they are anxious to try an FDA-approved product.
Zaki's story and several others led to global attention, and even a mass relocation to Colorado by parents across the globe seeking treatment for their children's seizures.
"Our families have been waiting for this exciting announcement", CEO of Epilepsy Foundation Central and South Texas, Sindi Rosales said.
The FDA's decision was not unexpected, as the proprietary extract formulation - developed by the British biotechnology firm GW Pharmaceuticals - had previously demonstrated safety and clinical efficacy at reducing seizure frequency in several placebo-controlled trials. Like other drugs in this category, which include heroin and cocaine, these drugs are considered to have no medical use and a high potential for abuse.
Epidiolex was recommended for approval by an advisory committee in April, and the agency had until this week to make a decision. The drug should be available in the coming months.
As a result, Epidiolex's launch remains at the discretion of the DEA, which must now evaluate the drug and consider reclassifying it as a substance that has medical properties, so as to allow GW to begin selling it.
The European Medicines Agency is now reviewing Epidiolex for treating seizures associated with LGS and Dravet Syndrome, with a decision on whether or not to recommend approval expected early next year. Though other drugs are approved to treat Lennox-Gastaut syndrome, Epidiolex is the first licensed to treat Dravet syndrome.
Shauna Garris, a pharmacist, pharmacy clinical specialist and adjunct assistant professor at the University of North Carolina's Eshelman School of Pharmacy, said the drug is effective and works somewhere between "fairly" and "very well". But FDA officials have said they believed the risks were manageable if cautionary language was placed in the drug's prescribing label, and if the agency monitored liver risk after the drug was on the market. Only products that have received formal FDA approval can make such claims, typically requiring clinical trials costing millions.
CBD does not cause intoxication or euphoria.
Epidiolex is based on cannabidiol, more commonly called CBD, rather than THC, which is the component associated with psychoactive effects.
Relying on homemade oil "is is a awful system, creating products that likely have more variability and can potentially contribute to more side effects and less consistency", Devinsky said.