FDA Approves Fenfluramine Drug Therapy for Dravet Syndrome

The Clinical and Translational Science Institute’s Clinical Research Services (CRS) program is excited to announce the FDA approval of the drug therapy Fintepla (fenfluramine) for Dravet syndrome, a severe form of pediatric epilepsy with frequent or prolonged seizures. CRS provided critical support for the drug research and trial over the past few years through its Pediatric Clinical Research Center (PCRC). This study has been active at UCSF since April 2016.


In the official FDA news release, Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research stated that “Dravet syndrome is a debilitating disease that takes a tremendous toll on both patients and their families. Fintepla offers an additional effective treatment option for the treatment of seizures associated with Dravet syndrome.”


The multicenter study, a randomized, double-blind, parallel Group, placebo-controlled trial titled, “Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults with Dravet Syndrome,” enrolled participants from ages to ages 2 to 18 who traveled from nearby San Francisco and as far as Southern California.­

The thoroughness and expertise of the entire CTSI CRS team and echo team were integral to the success of this protocol here at UCSF. It was a complicated protocol to learn and coordinate...

Lucy Liu, Senior Clinical Research Coordinator

UCSF Neurology & Pediatric Epilepsy


Each clinic visit collected more than 30 measures, including common ones such as vital signs to uncommon ones such as echocardiograms. The study team worked closely with the PCRC nursing staff, core lab, investigational drug services (IDS), and pediatric echo teams for hundreds of clinical visits. After the double-blind protocol, the study team continued working with these families through open-label extensions - the study’s principal investigator, Joseph Sullivan, MD, professor of neurology, has been working with some families on the trial for over four years now.


“With the overwhelming majority of our subjects traveling many hours to our hospital, it has always been important to conduct each site visit both thoroughly and correctly to avoid burdening families with an additional trip between scheduled study visits,” said Lucy Liu, MS, Senior Clinical Research Coordinator in neurology and pediatric epilepsy.


“From the time I joined the team in January 2018 to just a year later, the flow of a clinic visit had vastly improved from 1.5 hours to just under an hour at the PCRC. The close coordination in scheduling PCRC appointments and echo appointments had brought the length of a study day at UCSF from 8 hours to 2 hours.”


Liu adds that “The thoroughness and expertise of the entire CTSI CRS team and echo team were integral to the success of this protocol here at UCSF. It was a complicated protocol to learn and coordinate, but I had the support of nurses, lab techs, pharmacist, and sonographers who kept the trial running even across the transition of CRCs.”


Liu also shared that the CRS staff were deeply involved in the interviews, tours, and documentation during the inspection by the European Medicines Agency for its new drug application (NDA). “I am deeply grateful for all of their hard work day after day, year after year.”


Going Forward


The study team is currently working with its sponsor to coordinate the process of transitioning study subjects from the trial to outpatient clinical care. The study team hopes the next steps will include looking into the drug's application in helping people with other types of epilepsy.


Read the FDA News Release here