Drug intelligence / Profile preview

fenfluramine

Development stage
Approved
Lead developer
UCB
Modality
Small Molecules
Administration
Oral
01

Overview

Fenfluramine is a small molecule anticonvulsant and serotonergic agent used primarily for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients aged two years and older. It was originally developed as an appetite suppressant but was withdrawn from the market due to cardiovascular toxicity, specifically valvular heart disease and pulmonary arterial hypertension. The drug acts by increasing extracellular serotonin levels through modulation of serotonin transporters, agonism at several serotonin receptors (notably 5‑HT1D, 5‑HT2A, 5‑HT2B, 5‑HT2C), and antagonism or positive modulation at sigma σ1 receptors. Its precise mechanism in seizure control is not fully understood but likely involves increased GABAergic neurotransmission via serotonergic pathways and sigma receptor modulation. Fenfluramine is currently marketed under the brand name Fintepla by UCB (formerly Zogenix) for epilepsy indications[1][2][3][4][6].

Brand names
FinteplaPondimin
Other names
(±)-fenfluramineDL-fenfluramineFenfluraminaFenfluraminum1-(m-trifluoromethyl-phenyl)-2-ethylaminopropane
02

Targets

HTR2C (5-hydroxytryptamine 2C receptor)HTR2A (Serotonin receptor 5-HT2A)SERT (Sodium-dependent serotonin transporter)SIGMAR1 (Sigma non-opioid intracellular receptor 1)

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