UCB’s Fintepla has been approved to treat seizures associated with two epilepsy disorders and now has posted data that could pave the way for its use in another epilepsy condition.
A phase 3 trial of Fintepla in patients with the ultra-rare CDKL5 deficiency disorder (CDD) has achieved its primary and most of its secondary endpoints, the Brussels-based company said in a June 27 press release.
The study enrolled 87 participants between the ages of 1 and 35 who have been diagnosed with CDD and uncontrolled seizures. In comparing the performance of Fintepla to placebo, the primary endpoint was based on the median percent change in countable motor seizure frequency between baseline and the titration-plus-maintenance trial phase. UCB said it will present the results at an upcoming medical conference.
In a statement, UCB's Fiona du Monceau, EVP of patient evidence, said the company looks forward to working with regulators to make the drug available "as soon as possible."
Fintepla was well-tolerated in the trial, with its safety profile consistent with previous studies. UCB is conducting a 54-week extension phase of the trial to investigate the long-term tolerability of the oral treatment.
The median age for CDD onset is 6 weeks, UCB said. The genetic condition causes neurodevelopmental delays resulting in intellectual, motor, cortical visual, and sleep impairments. It is estimated that CDD affects approximately 1 in 40,000 to 60,000 live births.
Fintepla was first approved in 2020 to treat seizures associated with Dravet syndrome, a rare, severe form of epilepsy. Two years later, Fintepla gained expansion into a larger epilepsy indication, Lennox-Gastaut syndrome, which affects between 1 to 2 million people worldwide.
Months before the Lennox-Gastaut nod, UCB paid $1.9 billion to acquire Fintepla and its developer, California-based biotech Zogenix. The deal was a strategic fit for UCB, which then had four epilepsy drugs on the market but was losing market exclusivity for the most successful one, Vimpat, a blockbuster that outsold the other three combined.
Fintepla appears to be on a blockbuster track of its own, with sales reaching 340 million euros ($368 million) last year, which was a bump from sales of 226 million euros ($243 million) in 2023. After a 2023 settlement in a patent dispute, UCB expects to retain U.S. exclusivity on Fintepla until 2033.