Clinical Trials Arena

UCB pursues approval of Fintepla in CDKL5 deficiency following Phase III study

The company has already secured approval for Fintepla in treating Dravet syndrome and Lennox-Gastaut syndrome.

UCB is seeking approval for its anti-seizure medication Fintepla (fenfluramine) after it achieved the primary and most secondary endpoints in a Phase III trial in patients with CDKL5 deficiency disorder (CDD).

While the company did not release any data, it confirmed that patients in the treatment arm met the primary endpoint of median percent change in countable motor seizure frequency (CMSF) between baseline and the titration plus maintenance phase.

The drug was well tolerated, with a safety profile like that seen in earlier studies, including trials of the drug in Dravet syndrome and Lennox-Gastaut syndrome.

Fiona du Monceau, executive vice president of Patient Evidence at UCB, said: "These results pave the way for creating significant therapeutic progress and represent an important milestone in UCB's mission to bring meaningful innovation to individuals and families affected by developmental and epileptic encephalopathies.”

The randomised, double-blind, placebo-controlled study examined the efficacy and safety of adjunctive Fintepla in 87 patients aged one to 35, with a CDD diagnosis and uncontrolled seizures.

UCB is now conducting an open-label, flexible-dose, 54-week extension phase of the study to characterise the long-term safety and tolerability of Fintepla in paediatric and adult patients with CDD.

CDD is an ultra-rare developmental and epileptic encephalopathy with refractory infantile-onset epilepsy and severe global neurodevelopmental delays resulting in intellectual, motor, cortical visual, and sleep impairments as major features.It is caused by pathogenic variants in the cyclin dependent kinase-like 5 (CDKL5) gene located on the X chromosome.

Monceau added that it is speaking with the health authorities to seek approval of Fintepla in CDD. In the US, Fintepla has been approved for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients two-years of age and older.It is not approved for use in CDD by any regulatory authority worldwide.

There is only one other therapy approved for CDD by the US Food and Drug Administration (FDA), Marinus Pharmaceuticals’ Ztalmy (ganaxolone). The drug was approved on 18 March 2022 for patients aged two and older.