FDA OKs New Treatment for Seizures Associated with Dravet Syndrome

Officials with the FDA approved fenfluramine (Fintepla, Zogenix) oral solution, CIV for the treatment of seizures associated with Dravet syndrome in patients 2 years of age and older, according to a press release.

Dravet syndrome is a rare, childhood-onset epilepsy characterized by frequent and severe treatment-resistant seizures, associated hospitalizations and medical emergencies, significant development and motor impairments, and an increase risk of sudden unexpected death. However, despite existing therapies, there remains an unmet need in Dravet syndrome to reduce convulsive seizures.

The approval of fenfluramine oral solution in Dravet syndrome is based on data from 2 randomized, double-blinded, placebo-controlled phase 3 clinical studies, and safety data from an open-label extension trial.

According to the results, fenfluramine significantly reduced the monthly convulsive seizure frequency when added to existing treatment regimens compared with a placebo in study patients whose seizures were not adequately controlled on 1 or more antiepileptic drugs. Additionally, most patients in the study responded to treatment with fenfluramine within 3 to 4 weeks and these effects remained consistent over the treatment period.

Overall, the most common adverse reactions were decreased appetite, somnolence, sedation, lethargy, diarrhea, constipation, abnormal echocardiogram, fatigue, malaise, asthenia, ataxia, balance disorder, gait disturbance, increased blood pressure, drooling, salivary hypersecretion, pyrexia, upper respiratory tract infection, vomiting, decreased weight, fall, and status epilepticus. 

“There remains a huge unmet need for many Dravet syndrome patients who continue to experience frequent severe seizures even while taking 1 or more of the currently available anti-seizure medications,” Joseph Sullivan, MD, director of the Pediatric Epilepsy Center of Excellence at the UCSF Benioff Children’s Hospital and the principal investigator, said in a statement. “Given the profound reductions in convulsive seizure frequency seen in the Fintepla clinical trials, combined with the ongoing, robust safety monitoring that will be part of its use, I feel Fintepla will offer an extremely important treatment option for Dravet syndrome patients.”

Fenfluramine for use in Dravet syndrome will be launched through a restricted distribution program, called the Fintepla Risk Evaluation and Mitigation Strategy (REMS) Program, and is expected to be available through Zogenix’s specialty pharmacy partner by the end of July.

Reference

FDA Approves FINTEPLA (fenfluramine) for the Treatment of Seizures Associated with Dravet Syndrome. News Release. Zogenix; June 25, 2020. Accessed June 26, 2020. https://zogenixinc.gcs-web.com/news-releases/news-release-details/fda-approves-finteplar-fenfluramine-treatment-seizures