In mid-October, biopharmaceutical company Zogenix announced that its Dravet syndrome treatment FINTEPLA (fenfluramine) received a positive CHMP opinion. The CHMP is part of the European Medicines Agency (EMA). While FINTEPLA received the positive opinion, no decision has yet been made on Zogenix’s Marketing Authorization Application. However, the EMA’s decision should occur before 2020 ends.
This orally administered prescription medicine is designed to treat seizures in patients ages 2 or older. In many cases, these young patients with Dravet syndrome often experience multiple symptoms even while on other anti-epileptic medicine. FINTEPLA is designed to be taken in conjunction with other medications. The therapy’s positive opinion is based on results from two Phase 3 clinical trials and one extension study. Through these trials, 330 patients enrolled. Data shows that FINTEPLA is relatively safe, effective, and well-tolerated. It reduced seizure frequency and sustained this reduction over a period of time. However, there were some side effects, which included:
- Appetite loss
- Nausea and diarrhea
- Fatigue and lethargy
- Increased drowsiness
First described by French psychiatrist and epileptologist Dr. Charlotte Dravet in 1978, Dravet syndrome is a rare genetic disorder causing severe epilepsy, which begins in infancy. The condition causes a genetic brain dysfunction called epileptic encephalopathy. An estimated 1 in 15,700-40,000 infants have Dravet syndrome. The disorder is lifelong, resulting in frequent and often treatment-averse seizures. Temperature changes, like a warm bath or fever, may trigger seizures. Initial seizures may only affect half of the body. Generally, Dravet syndrome affects males and females equally. For an estimated 10-20% of patients, Dravet syndrome is fatal before adulthood.
- Long-lasting seizures
- Impaired balance and coordination
- Developmental and cognitive delays
- Gait abnormalities
- Low muscle tone
- Behavioral difficulties
- Sudden unexpected death in epilepsy (SUDEP)
- Status epilepticus, or a state of prolonged/continuous seizure that requires immediate medical attention
Learn more about Dravet syndrome here.