US FDA gives green light to UCB’s drug for rare childhood epilepsy

The FDA also granted paediatric exclusivity to the oral solution, branded Fintepla.  

The oral solution Fintepla (fenfluramine) was already approved in the US and EU for the treatment of seizures associated with Dravet syndrome in patients two and older. It will be available for treatment of Lennox-Gastaut syndrome (LGS) in the US through a restricted distribution programme.  

LGS is a severe form of childhood epilepsy that is characterised by multiple types of seizures, cognitive dysfunction, impaired motor function, and in some cases sudden unexpected death in epilepsy (SUDEP). UCB's Zogenix unit estimates that about 30,000 to 50,000 people have LGS in the United States.  

The FDA approval was supported by safety and efficacy data from a global, randomised, placebo-controlled Phase 3 clinical trial in 263 patients with LGS. 

Fintepla’s approval comes just a few weeks after UCB closed its acquisition of biopharmaceutical firm Zogenix, who developed the drug, in a deal valued at up to $1.9 billion. Fintepla is also being targeted as a treatment for a genetic epilepsy called CDKL5 deficiency disorder in a Phase 3 trial. Before the acquisition, Zogenix stated that the drug could reach $1 billion in peak sales. 

Fintepla adjusts a receptor called Sigma 1, which controls how brain cells communicate with each other. It comes with a boxed warning for heart and lung conditions such as valvular heart disease and pulmonary arterial hypertension. 

UCB’s Fintepla will now be in direct competition with Jazz Pharmaceuticals’ cannabis-based Epidiolex, which was approved to treat seizures related to Dravet Syndrome and LGS in 2018. Other drugs with a similar offering to Fintepla include Takeda Pharmaceutical's soticlestat and Eisai’s lorcaserin and Fycompa.