The U.S. Food and Drug Administration (FDA) has expanded the age range of patients to whom Epidiolex (cannabidiol) can be prescribed, to include children as young as age 1, according to its developer, GW Pharmaceuticals (GW).
The medication had previously been made available to patients ages 2 and older who experience seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome.
In addition to LGS and Dravet, the FDA has approved Epidiolex to treat seizures associated with tuberous sclerosis complex, a rare genetic disorder in which epilepsy results from the growth of benign tumors.
“This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines,” Justin Gover, CEO at GW, said in a press release.
“It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug,” headded.
Made from cannabidiol or CBD, Epidiolex is the first plant-derived cannabinoid prescription medicine of its kind. It also is the only FDA-approved formulation of CBD.
CBD is a non-psychoactive component of the cannabis plant. It mimics certain natural compounds that can interact with brain receptors that, when defective, can trigger seizures.
Although its exact mechanism of action remains unknown, some evidence suggests that it regulates the endocannabinoid system, found within the brain and spinal cord, which controls the release of certain hormones and the activity of nerve cells.
Researchers believe that CBD also may regulate calcium levels, which are important to healthy communication between nerve cells.
Because CBD does not have the psychoactive, or mind-altering effects characteristic of delta (9)-tetrahydrocannabinol (THC) — the primary active component of cannabis — the U.S. Drug Enforcement Administration has classified Epidiolex as a non-controlled substance. Therefore, the enforcement agency has removed all federal controlled-substance restrictions previously placed on CBD, giving patients easier treatment access.
The results of three Phase 3 clinical trials — NCT02091375, NCT02224703, and NCT02224560 — contributed to Epidiolex’s FDA approval. These trials and an open-label extension study (NCT02224573) demonstrated that cannabidiol was both safe and effective as an add-on epileptic treatment in patients with LGS and Dravet.
Within the U.S., Epidiolex is marketed by Greenwich Biosciences, a subsidiary of GW. In Europe, the EU has approved Epidiolex under the trade name Epidyolex.