Treatment for ultra-rare form of epilepsy shows promising results

A late-stage study has shown promising results for the effectiveness of fenfluramine in people with an ultra-rare form of epilepsy - CDKL5 deficiency disorder (CDD).

CDD affects approximately one in 40,000 to 60,000 live births. The disorder causes uncontrolled seizures and symptoms usually appear at six weeks of age, alongside learning and developmental issues.

Fenfluramine, marketed under the brand name Fintepla, already holds approvals to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two other rare and severe forms of epilepsy.

The drug was found to be well tolerated by the people participating in the trial, aged from one to 35 years of age. Its safety profile was consistent with previous studies in two other rare epilepsies, Dravet and Lennox-Gastaut syndromes.

Now the pharmaceutical company UCB is conducting a further 52-week extension phase of the study to assess the long-term safety of fenfluramine in adult and paediatric CDD patients.

UCB said it is planning to submit regulatory applications for fenfluramine in CDD to bring the drug to patients “as soon as possible”.