Rare new drug for childhood epilepsy! FDA approves Fintepla (fenfluramin oral solution) for the treatment of Dravet syndrome!

, June 25, 2020 /
PRNewswire/ -- Zogenix is a pharmaceutical company dedicated to the development of treatments for rare diseasesRecently, the company announced that the U.SFood and Drug Administration (FDA) has approved Fintepla (fenfluramine, fenflulamine) oral solution CIV for use in patients up to 2 years of age to treat epilepsy associated with Dravet syndromeThe drug was approved through a priority review process afterFDAhas granted Fintepla the Orphan Drug (ODD) and Breakthrough Drug (BTD) for the treatment of Dravet syndrome-related epilepsyDravet syndrome is a rare childhood epilepsy characterized by frequent and severe drug-resistant seizures, associated hospitalizations and medical emergencies, severe developmental and motor disorders, and an increased risk of sudden accidental death (SUDEP)Fintepla is a low-dose fenfluraminin for liquid formulations that can reduce the frequency of seizures by regulating serotonin receptors and sigma-1 receptor activity (see Reference:)Fenfluraminees NMDA oedd-mediated svia its mixed activity at serotonin 5HT2A and type 1 sigma seros)data from two placebo-controlled Phase IIIclinical trialsshowed that fintepla significantly reduced the frequency of seizures compared to placebos in patients whose seizures were not adequately controlled by other drugs"For many patients with Dravet syndrome, there is still a huge need that remains unmet, even if they are taking one or more of the anti-epileptic drugs currently available, still have frequent severe seizures," said DrJoseph Sullivan, lead investigator at Fintepla Clinical Research at the University of California, San Francisco, and director of the Center for Child Epilepsy Excellence at Benioff Children's Hospital at the University of California, San FranciscoGiven Fintepla's significant reduction in the frequency of convulsive seizures inclinical trials, coupled with continuous and robust safety monitoring, I believe that Fintepla will provide an extremely important treatment option for patients with Dravet syndrome"
fenfluramin-molecular structure (photo: Wikipedia.org)FDAapproved Fintepla to treat Dravet syndrome-related epilepsy, based on data from two randomized, double-blind, placebo-controlled phase IIIclinical trials(conducted in patients aged 2-18 and published in Lancet and JAMA Morgan, respectively), and an open-label extension trial (many patients receiving Fintepla treatment for up to 3 years)The data showed that in study patients who took one or more anti-epileptic drugs that did not adequately control seizures, Fintepla significantly reduced the frequency of monthly seizures (convulive seizures, CS) compared to placebos based on existing treatment optionsIn addition, most of the study patients responded within 3-4 weeks of initiating Fintepla therapy and maintained consistent results throughout the treatmentnoteworthy, Fintepla's drug label includes a black-box warning that suggests the drug is associated with heart valve disease (VHD) and pulmonary arterial hypertension (PAH)Because of these risks, patients must undergo an echocardiogram monitoring every six months before and during treatment, and an echocardiogram monitoring every three to six months after the end of treatmentIf an echocardiogram shows signs of heart valve disease, PAH, or other heart abnormalities, health care professionals must consider the benefits and risks of continuing to treat patients with FinteplaDue to VHD and PAH risk, Fintepla can only be used under a restricted drug distribution program under risk assessment and mitigation strategies (REMS) Fintepla REMS requires healthcare professionals who prescribe Fintepla and pharmacies that distribute Fintepla to be specifically certified in Fintepla REMS and requires patients to register with REMS As part of the REMS requirements, prescribing physicians and patients must follow the required echocardiogram heart monitoring in order to receive Fintepla treatment in clinical studies, Fintepla treated the most common adverse reactions
(incidence of more than 10%, and higher than placebo) included: decreased appetite, drowsiness, sedation and drowsiness, diarrhea, constipation, echocardiogram abnormalities, fatigue or fatigue, co-operative disorders, balance disorders, gait disorders, elevated blood pressure, drooling, saliva secretion, fever, upper respiratory tract infections, vomiting, weight loss, and persistent epilepsy in addition to Dravet syndrome, Zogenix is also developing Fintepla 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 of onset, diverse types of seizures, high frequency of seizures, serious damage to intelligence, and difficult to treat In the United States, Fintepla was granted breakthrough drug (BTD) for the treatment of Dravet syndrome, orphan medicine (ODD) for the treatment of Dravet syndrome and LGS (BioValleyBioon.com) original source: Zogenix .