The treatment is a purified, CBD-rich cannabis extract marketed under the name Epidiolex. Pre-clinical research and anecdotal reports suggest that the CBD compound in marijuana has significant anti-seizure properties.
On top of its orphan drug status, Epidiolex was recently approved by the FDA as an investigational new drug (IND), which has allowed independent U.S. specialists to begin treating high-need children with the new drug immediately.
According to Friday’s press release, a total of seven “expanded access” IND applications for Epidiolex have been granted, allowing doctors to treat approximately 125 children with various forms of epilepsy, including Dravet syndrome, Lennox-Gastaut syndrome and others.
The company says the INDs will provide observational data that will aid in the clinical development process.
Dr. Orrin Devinsky, a professor at NYU School of Medicine and one of the specialists selected to trial Epidiolex, expressed optimism for the treatment.
“I, together with many colleagues in the U.S. who specialize in the treatment of childhood epilepsy, very much welcome the opportunity to investigate Epidiolex in the treatment of Dravet syndrome. The FDA’s timely approval of the orphan drug designation for Epidiolex in Dravet syndrome is a key milestone that comes after many years of reported clinical cases that suggest encouraging evidence of efficacy for CBD in this intractable condition.”
GW Pharmaceuticals Chief executive Justin Gover adds that receiving orphan drug status was an important “corporate strategic priority,” since it offers financial incentives for developing treatments for rare diseases.
Both Dravet syndrome and Lennox-Gastaut syndrome are rare, severe forms of pediatric epilepsy.
U.S. federal law classifies marijuana as having no recognized medical use Interest in marijuana-based treatments peaked this summer after CNN aired the story of Charlotte Figi – a six-year-old girl with Dravet syndrome whose family found CBD-rich cannabis to be the only treatment that worked.
However, marijuana remains prohibited under federal law, classified as a schedule 1 substance with no recognized medical use, leaving patients in many states without a way of accessing the treatment.
On the other hand, GW Pharmaceuticals has become a leading manufacturer of cannabis-based drugs, holding a special permit from the UK government to grow cannabis. The company is best known for manufacturing Sativex, an oral spray with an equal 1:1 ratio of THC and CBD.
Sativex has been approved in over 20 countries for the treatment of multiple sclerosis symptoms, and is currently been studied as a treatment for cancer pain as well as aggressive brain cancer.
While Sativex has yet to be approved in the U.S., the approval process for Epidiolex could be sped up if early results are promising.