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FDA Backs Cannabis-Based Drug to Treat Kids With Epilepsy


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The mission of the U.S. Food and Drug Association (FDA) is to protect the public’s health in relation to drugs, biological products and medical devices sold within the U.S.1 When a pharmaceutical company is on the pathway to the approval of a drug, the FDA utilizes a process which may or may not require the use of an Advisory Committee. According to the FDA’s website:

“When a scientific, technical, or policy question arises … FDA often relies on Advisory Committees to provide independent advice.”2

Upon the organization of an Advisory Committee, a full-time FDA employee will act as chairperson. However, many or most of the committee’s members will represent third-party interests, including scientific expertise, industry and potential future patients. All selected committee members and their respective knowledge and experience reflect the questions and concerns FDA has in regard to the drug in question.

Patients and clinical trial participants who have taken the scrutinized drug experimentally are also invited to participate, more often than not. These participants provide a real-world view that can become influential to the FDA’s final decision.3 Real-world participants are given a chance to address the committee and any members of the public interested in the discussion at hand.4 As with many official gatherings, it isn’t uncommon for the chairperson to ask that any applause be held until all participants have had their chance to speak.

However, in one of the more recent Advisory Committee meetings, held on April 19, a 16-year-old boy stood up to address committee members and the public in attendance. After he spoke, the audience ignored protocol and applauded the teenager’s words. Sam Vogelstein, a high-school student and epilepsy patient, was the first U.S. citizen to try the experimental drug, Epidiolex. According to the FDA, Epidiolex is a “priority review” drug because of its success in treating disease with few or no alternative treatments. Priority review status ensures that a drug will be reviewed in six months instead of the standard 10.5

Today, Sam still takes the drug and has a lot to say about his experiences:

“My parents tell me there were times I had seizures 100 times a day.”

“I can understand what goes on at school [now that I am taking Epidiolex].”

“… I can have adventures that never would have been possible before.”6

After the proceedings, USA Today reported that the committee’s “13-member FDA panel voted unanimously in favor of the experimental medication [Epidiolex] made from a chemical found in cannabis – one that does not get users high.7 The panelists backed the drug based on three studies showing that it significantly reduced seizures in children with two rare forms of childhood epilepsy: Dravet and Lennox Gastaut syndromes. 


Marci Crane is a staff writer for GxP Lifeline and has written on life science topics for over a decade.


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