US FDA panel recommends approval of cannabis-derived drug for the first time
Epidiolex may soon become the first cannabidiol available on prescription in the United States, after a US Food and Drug Administration expert panel voted to recommend it for the treatment of two rare forms of epilepsy.
A US Food and Drug Administration (FDA) expert panel has voted in favour of approving the cannabinoid medication Epidiolex (cannabidiol) for the treatment of seizures in two rare forms of epilepsy.
The oral solution contains plant-based purified cannabidiol, a chemical component of marijuana that lacks the psychoactive effects associated with the drug. The FDA panel has recommended its approval as an add-on therapy for patients aged two years and over with seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.
Both conditions emerge during early childhood and are frequently resistant to existing medications. Patients with Lennox-Gastaut syndrome (LGS) experience drop seizures — a sudden loss of muscle tone that can lead to falls and injury. And seizure frequency in Dravet syndrome has been associated with developmental delay and the risk of potentially fatal status epilepticus.
A recent phase III study, published in the Lancet, showed a 43.9% reduction in drop seizures after 14 weeks of treatment in patients with treatment-resistant LGS. Meanwhile, a trial published in the The New England Journal of Medicine reported a decrease in the median frequency of convulsive seizures per month, from 12.4 to 5.9 in patients with Dravet syndrome.
“We are pleased by the Advisory Committee’s unanimous recommendation to approve Epidiolex, which would provide an important treatment option for patients with LGS and Dravet syndrome —two of the most severe and treatment-resistant forms of epilepsy,” said Justin Gover, chief executive officer at GW Pharmaceuticals, the British company that developed Epidiolex.
Although some US states have made provision for the availability of legal medical marijuana, this will become the first cannabis-derived prescription drug to be approved by the FDA, if full approval follows. A decision is expected in the summer. Other cannabis-based treatments available in the United States, such as those for the treatment of chemotherapy-related side effects, are synthetically made. In the UK, the plant-based Sativex (delta-9-tetrahydrocannabinol and cannabidiol), which was also developed by GW Pharmaceuticals, received a licence for the improvement of spasticity in people with multiple sclerosis in 2010.
Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2018.20204763
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