What does a PDUFA date mean for cannabidiol (CBD)?

What is a PDFUA date?

On December 28, 2017, a Prescription Drug User Fee Act (PDUFA) date was released by the U.S. Food and Drug Administration (FDA) for Epidiolex^®. Epidiolex is an investigational, plant-based purified cannabidiol (CBD) medication. This product was submitted to the FDA for potential approval as a treatment option for epilepsy patients with Dravet syndrome or Lennox- Gastaut syndrome (LGS).

A PDUFA date serves as a goal date for the FDA to make a decision about whether or not to approve new medications. The FDA is normally given 10 months to review a new drug after the filing application is accepted. If a drug is selected for priority review, this means that the FDA’s goal is to take action on an application within 6 months (compared to 10 months under standard review).

History of PDUFA

PDUFA refers to a law initially passed by the United States Congress in 1992 that created the FDA user fee program and allowed the FDA to collect fees from drug manufacturers at the time of a New Drug Application (NDA). The fees fund a new drug approval process due to the costs associated with such a process. The user fee programs have been reauthorized several times since 1992, with the latest reauthorization, PDUFA VI, in 2017. The user fee program ensures FDA can sustain its important work and continue to develop and refine its processes to get more valuable therapies to the market in a timely manner.

As part of the PDUFA agreements, FDA collects fees in exchange for meeting certain requirements that are primarily related to:

• The speed of certain activities within the NDA review process
• The announcement of a date by which a review should be completed
• Additional initiatives that incorporate the patient voice and perspective to the drug review process

What does the PDUFA date mean for Epidiolex?

For Epidiolex, the PDUFA goal date is June 27, 2018, which means this drug was given Priority Review status (a shortened, 6-month review period). Priority review status is designated for drugs that may offer major advances in treatment or provide a treatment where no adequate therapies exist. In its acceptance letter, the FDA has stated that it is currently planning to hold an advisory committee meeting to discuss this application.

What does this mean for people with Dravet syndrome and LGS?

• This means that a decision by FDA is likely to be made this summer to determine if Epidiolex will be FDA approved for the treatment of Dravet syndrome and LGS.
• If Epidiolex is approved by the FDA, the U.S. Drug Enforcement Agency (DEA) will have 90 days to take action by scheduling the medication or asking for additional review time.
• Because Epidiolex is derived from a Schedule I substance, states will have to take legislative or regulatory action for the medication to become available in the state.
• Once Epidiolex is scheduled by DEA and states takes action to create a pathway for the medication to enter the market, it can become available in pharmacies for providers with a regular medical prescribing license to write a prescription for patients the FDA approval covers.
• For those waiting for a possible CBD treatment that has been studied properly and regulated for consistency, there is “a light at the end of the tunnel.”

Editor’s Note: Through our targeted research initiative and new therapy grants program, the Epilepsy Foundation funds research to investigate cannabinoids as a treatment for epilepsy. Find out how your gift matters and how you can support the Epilepsy Foundation.

Learn More

• Our Work on State Rescheduling for FDA-approved Therapies Derived from CBD
• Learn more about PDUFA and the user fee programs at FDA
• About Medical Marijuana and Epilepsy
• A Parent's Perspective: the LGS Epidiolex Study (article January 2018)
• New England Journal of Medicine Publishes Data on Purified CBD, Epidiolex, for People with Dravet Syndrome (article, May 2017)