Rare new drug for children with epilepsy! Fintepla (fenfluoramine oral solution) is recommended by the EU CHMP for the treatment of Dravet syndrome!

October 19, 2020 // -- Zogenix is a pharmaceutical company dedicated to the development of drugs for the treatment of rare diseases.
recently, the company announced that the European Medicines Agency (EMA) Human Pharmaceutical Products Committee (CHMP) has issued an active review recommending approval of Fenfluramine oral solution as an additional (add-on) therapy for other anti-epileptic drugs for patients ≥2 years of age with Dravet syndrome.
CHMP opinion is the final step in the marketing authorization process before obtaining approval from the European Commission (EC).
, CHMP's comments will now be submitted to the EC for review, which usually takes CHMP's advice and is expected to make a final review decision by the end of the year.
U.S., Fintepla was approved in June to treat≥ epilepsy associated with Dravet syndrome in patients as young as 2 years old.
the drug was approved through a priority review process after the FDA granted Finetepla the orphan drug (ODD) and breakthrough drug eligibility (BTD) for the treatment of Dravet syndrome-related epilepsy.
Dravet syndrome is a rare childhood epilepsy characterized by frequent and severe drug-resistant seizures, associated hospitalization and medical emergencies, severe developmental and motor disorders, and an increased risk of sudden accidental death (SUDEP).
Fintepla is a liquid preparation with a low dose of fenfluramin that reduces the frequency of seizures by regulating serotonin receptor and sigma-1 receptor activity (see reference: Fenfluramine designes NMDA receptor-mediateded via its mixed activity at serotonin 5HT2A and type 1 sigma receptors).
from two placebo-controlled Phase III clinical trials, Fintepla significantly reduced the frequency of convulsive seizures compared to placebos in patients with other drugs that did not adequately control seizures.
. Stephen J. Farr, President and CEO of Zogenix, said, "We are pleased that CHMP has conducted a regulatory review of the quality, safety and effectiveness of Findepla and has given a positive review.
Six years ago, after researchers in Belgium realized the potential of fenfluoramine, a pharmacological drug with different pharmacological effects than other anticonvulsant drugs, to treat Dravet syndrome's persistent epilepsy, we embarked on Fintepla's rigorous global development program.
many people with Dravet syndrome experience frequent and severe seizures even when taking one or more of the anti-epileptic drugs currently available.
for this reason, we are pleased to bring Fintepla as an important new treatment to Dravet syndrome patients and their families in Europe.
, director of paediatric neurology at the University Hospital of Leuven in Belgium, said: "Reducing the frequency of seizures is the first and most important step in treating all children with Dravet syndrome.
I am pleased that in all Phase 3 studies, fenfluoramine therapy has reduced the frequency of seizures in patients with Dravet syndrome clinically and statistically significantly.
" fenfluoramine-molecular structure (photo source: Wikipedia.org) CHMP's positive opinion is based on data from two randomized, double-blind, placebo-controlled Phase III clinical trials (conducted in patients aged 2-18 years, published in Lancet and JAMA Neurology, respectively) and security data from an open label extension trial in which many patients were treated with Findepla for up to three years.
data show that fintepla significantly reduced the frequency of monthly convulsive seizures (convulsive seizures, CS) compared to placebos in patients who took one or more anti-epileptic drugs that did not adequately control seizures.
, most of the patients in the study responded within 3-4 weeks of initiating The Findepla treatment and had consistent results throughout the treatment.
, the most common adverse events in these studies included diarrhea, fever, fatigue, upper respiratory tract infections, drowsiness, and bronchitis.
no patients experienced any adverse cardiovascular events, including valve heart disease or pulmonary hypertension.
addition to Dravet syndrome, Zogenix is also developing Findepla to treat seizures associated with Lennox-Gastaut syndrome (LGS).
Dravet syndrome and LGS are 2 rare and often catastrophic childhood seizures with early onset age, diverse types of seizures, high frequency of seizures, serious damage to intelligence, difficult to treat and so on.
() Origin: Zogenix Receives Positive CHMP Opinion for FINTEPLA® Oral Solution for Treatment of Patients with Dravet Syndrome