Zogenix, a global biopharmaceutical company developing rare disease therapies, announced that the Committee for Medicinal Products for Human Use (CHMP), a part of the European Medicines Agency (EMA), has adopted a positive opinion recommending the marketing authorization of FINTEPLA (fenfluramine) oral solution for the treatment of seizures associated with Dravet syndrome, a rare and devastating infant- and childhood onset epilepsy, as an add-on therapy to other antiepileptic medicines for patients two years of age and older. The European Commission (EC) is expected to make a final decision on the company’s Marketing Authorization Application (MAA) by the end of the year.

“We are pleased that the CHMP’s regulatory review of FINTEPLA for quality, safety, and efficacy has resulted in their positive opinion,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix. “We began our rigorous global development program for FINTEPLA nearly six years ago after researchers in Belgium recognized the potential of fenfluramine, a drug with distinct pharmacology from all other anticonvulsant agents, to treat intractable seizures in Dravet syndrome. Many Dravet syndrome patients continue to experience frequent severe seizures even while taking one or more currently available anti-seizure medications. For this reason, we are excited to be another step closer to potentially introducing FINTEPLA as an important new treatment option for these patients and their families in Europe.”

“Reducing seizure frequency is the first and most important step in treating all Dravet syndrome children, regardless of age,” said Lieven Lagae, M.D, Ph.D., Full Professor and Head of Pediatric Neurology Department at the University Hospitals of Leuven in Belgium. “I am thrilled that all Phase 3 studies with fenfluramine demonstrated a clinically meaningful, highly statistically significant decrease of seizure frequency in Dravet syndrome patients.”

The MAA for FINTEPLA included positive results from two randomized, controlled Phase 3 trials (Study 1 and Study 2), together with an interim analysis of an ongoing long-term, open-label extension study involving a total of 330 Dravet syndrome patients. These studies demonstrated that adjunctive fenfluramine treatment provided a highly statistically significant and clinically meaningful reduction in convulsive seizure frequency compared to placebo and was generally well-tolerated. In one of the trials, all subjects were treated with a background regimen that included stiripentol, with significant improvement observed for FINTEPLA over placebo. The long-term, open-label extension study demonstrated durable efficacy, with patients in that study treated for up to three years with FINTEPLA. The most commonly reported adverse events experienced during these studies were decreased appetite, diarrhea, pyrexia, fatigue, upper respiratory tract infection, lethargy, somnolence and bronchitis. No patient developed any cardiovascular adverse events, including valvular heart disease or pulmonary arterial hypertension.

If authorized by the EC, FINTEPLA will be approved for use by patients with Dravet syndrome aged two years and older in all European Union member states, as well as the United Kingdom, Iceland, Liechtenstein and Norway. The product is expected to be made available under a controlled access program to ensure regular cardiac monitoring and to mitigate potential off-label use for weight management.

Earlier this year, FINTEPLA was approved by the U.S. Food & Drug Administration (FDA) for the treatment of seizures associated with Dravet syndrome in patients aged two years and older. A third positive Phase 3 clinical trial (Study 3) was recently reported to support registration of FINTEPLA in Japan.

Dravet syndrome is a rare, devastating and life-long form of epilepsy that begins in infancy and is marked by frequent, treatment-resistant seizures, significant developmental, motor, and behavioral impairments, and an increased risk of sudden unexpected death in epilepsy. Affecting one in 15,700 individuals in the U.S. and approximately one in 20,000 to 40,000 in Europe, most patients follow a course of developmental delay with cognitive, motor and behavioral deficits that persist into adulthood. Dravet syndrome severely impacts quality of life for patients, families and caregivers due to the high physical, emotional, and financial burden associated with the disease.