UCB's Fintepla Shows Promise in Third Epilepsy Indication

UCB's anti-epileptic drug Fintepla has demonstrated positive results in a phase 3 trial for CDKL5 deficiency disorder (CDD), potentially paving the way for its use in a third epilepsy indication. The Brussels-based pharmaceutical company announced on June 27 that the study achieved its primary endpoint and most secondary endpoints, marking a significant milestone in the treatment of this ultra-rare genetic condition.

Fintepla's Expanding Epilepsy Portfolio

Fintepla, initially approved in 2020 for Dravet syndrome, has been steadily increasing its reach in the epilepsy market. In 2022, it gained approval for Lennox-Gastaut syndrome, a more prevalent form of epilepsy affecting 1 to 2 million people worldwide. The latest trial results for CDD could further solidify Fintepla's position as a versatile anti-epileptic medication.

The phase 3 trial enrolled 87 participants aged 1 to 35 with CDD and uncontrolled seizures. The primary endpoint focused on the median percent change in countable motor seizure frequency between baseline and the titration-plus-maintenance trial phase, comparing Fintepla to placebo. While specific data were not disclosed, UCB plans to present the results at an upcoming medical conference.

CDD: A Rare and Challenging Disorder

CDKL5 deficiency disorder is an ultra-rare genetic condition with an estimated incidence of 1 in 40,000 to 60,000 live births. The disorder typically manifests around six weeks of age and causes severe neurodevelopmental delays, affecting intellectual, motor, and visual functions, as well as sleep patterns.

Fiona du Monceau, EVP of patient evidence at UCB, expressed the company's eagerness to work with regulators to make Fintepla available to CDD patients "as soon as possible." The drug's safety profile in the CDD trial was consistent with previous studies, and UCB is conducting a 54-week extension phase to investigate long-term tolerability.

Financial Implications and Market Outlook

UCB's acquisition of Fintepla and its developer, Zogenix, for $1.9 billion in 2022 appears to be paying off. The drug has shown strong sales growth, reaching 340 million euros ($368 million) in 2024, up from 226 million euros ($243 million) in 2023. With U.S. exclusivity expected to last until 2033 following a patent dispute settlement, Fintepla is on track to achieve blockbuster status.

The expansion into CDD treatment could further boost Fintepla's market potential, solidifying UCB's position in the epilepsy drug market. This is particularly significant as the company faces the loss of market exclusivity for its blockbuster drug Vimpat, which has been a cornerstone of UCB's epilepsy portfolio.