Rare new drug for children with epilepsy! Fintepla is approved by the European Union to treat patients with ≥-year-old Dravet syndrome!

December 24, 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 Commission (EC) has approved Fenfluramine oral solution as an additional (add-on) therapy for other anti-epileptic drugs for patients aged ≥2 years with Dravet syndrome≥
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 lifelong epilepsy that begins in infraction and is characterized by severe incurable seizures, frequent medical emergencies, severe cognitive and behavioral disorders, and an increased risk of sudden and unexpected death (SUDEP).
many people with Dravet syndrome experience frequent and severe seizures even when taking one or more of the anti-epileptic drugs currently available.
reducing the frequency of seizures is the first and most important step in treating all children with Dravet syndrome.
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).
drug has a pharmacological effect that differs from other anticonvulsant drugs.
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.
-Finfluoramine-Molecular Structure (Photo: Wikipedia.org) this EU-approved data 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 a long-term open label extension trial in which many patients were treated with Findepla for up to three years.
results showed 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 two rare and often catastrophic childhood seizures, characterized by early onset, diverse types of seizures, high frequency of seizures, severe damage to intelligence, and difficulty in treatment.
origin: The European Commission approves Zogenix's FINTEPLA® Oral Solution for Treatment of Dravet Syndrome