FDA Approval of the First Plant-Derived Cannabinoid Prescription Medication

In a landmark approval on June 25th, the Food and Drug Administration (FDA) approved Epidiolex® (oral cannabidiol solution) for the treatment of seizures associated with Lennox-Gestaut Syndrome or Dravet Syndrome, in children two years of age and older.  Epidiolex is the first marijuana-based drug approved for use in the United States. Two other drugs previously approved by the FDA – Marinol and Cesamet – are synthetic cannabinoids rather than naturally occurring components of the marijuana plant.  Epidiolex is also the first drug approved for the treatment of Dravet Syndrome. Lennox-Gestaut Syndrome and Dravet Syndrome are severe forms of childhood epilepsy.

While the drug’s current FDA approved indications would impact only a small number of workers’ compensation claims, the possibility of off-label use as further outlined below could have a significant impact on the workers’ compensation arena.

The FDA’s approval was based on outcomes data from three clinical trials, conducted with a total of 516 patients over fourteen weeks. Two of the studies were conducted with patients afflicted with Lennox-Gestaut Syndrome. Epidiolex was used as adjunctive therapy at a dose of 20mg per kg per day. In those studies, the investigators observed a statistically significant reduction in the frequency of drop seizures. The third study was conducted in patients with Dravet Syndrome, where the primary efficacy measure was the change in frequency of convulsive seizures. Within the third study population, a statistically significant reduction in the frequency of convulsive seizures was observed.  The most common adverse reactions observed in clinical trials included liver toxicity, anemia and drowsiness.  Upon approval, Epidiolex was classified as a schedule I due to marijuana’s classification as a schedule I controlled substance. According to federal regulatory guidelines, schedule I controlled substances have no currently accepted medical use in the United States, a lack of accepted safety for use under medical supervision and a high potential for abuse. Since Schedule I controlled substances cannot be legally dispensed in the United States, Epidiolex is expected to be rescheduled prior to its market launch in the fall of 2018. Industry experts are expecting the drug to be classified as a schedule IV or schedule V controlled substance since Epidiolex contains very little tetrahydrocannabinol (THC), which is the psychoactive component of marijuana.

The manufacturer of Epidiolex is GW Pharmaceuticals, a British drug manufacturer that specializes in the development of marijuana-based drugs. Another product manufactured by GW Pharmaceuticals is Sativex, an oral spray that contains both cannabidiol and THC. Sativex is approved in Europe, Canada, Australia, New Zealand and several other countries for the treatment of muscle spasticity caused by multiple sclerosis.  In Israel, Sativex is also approved for the treatment of pain and chronic non-cancer pain. In the Unites States, many states have expressly allowed the use of medical marijuana for various pain conditions. This certainly leaves the door open to the possibility that Epidiolex will be utilized in an off-label capacity for chronic pain and as a result, could have an impact in the workers’ compensation arena. The average wholesale price or AWP for Epidiolex has not been determined, so the cost of a typical regimen is unknown at this time.

For additional information regarding Epidiolex or other cannabinoid derived products, please email ComplexClaims@medval.com.

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