First dual-action anti-seizure drug PBS listed in milestone for UCB

Australians living with Dravet syndrome have access to a reimbursed dual-mechanism therapy that tackles seizures resistant to conventional treatments. UCB’s Fintepla (fenfluramine), now listed on the Pharmaceutical Benefits Scheme (PBS), is available as an add-on therapy for patients ≥2 years of age taking at least two other anti-epileptic drugs.

Professor Ingrid Scheffer AO, Chair of Paediatric Neurology Research at the University of Melbourne, said “Fenfluramine significantly reduces seizures in patients with Dravet syndrome. Controlling seizures in Dravet syndrome is critical as this may lead to improvements in development and other morbidities such as sleep, and even reduce the mortality risk.

“I am optimistic that fenfluramine will offer a step forward in the evolution of treatment for Dravet syndrome. It is wonderful that all families will be able to access this important treatment option through our health system.”

A recent review of UCB’s clinical trial data found that adjunctive treatment with Fintepla reduced the monthly convulsive seizure frequency (MCSF) by >50% to >60% vs placebo in patients with Dravet Syndrome whose seizures were inadequately controlled by their existing regimen that contained or did not contain stiripentol, respectively. Longer term, Fintepla provided sustained, clinically meaningful reductions in the MCSF in patients with Dravet Syndrome for up to 3 years.

Initially developed as an appetite suppressant and later withdrawn from the market due to cardiovascular risks, Fintepla was repurposed for Dravet syndrome patient cohort.

Professor Piero Perucca, Professor of Adult Epilepsy at Austin Health, emphasised the importance of this new therapy option in managing the condition across the lifespan.

“For individuals with Dravet syndrome, seizures evolve over time with a change in seizure type and frequency from childhood to adulthood. For clinicians, monitoring these changes it is critical to continue to assess the course of treatment.

“Greater seizure reduction is also associated with significant improvements in non-seizure outcomes such as emotion and cognitive regulation to improve quality of life for patients and carers. I welcome the reimbursement of fenfluramine. Through more access to innovative treatment options and continued research, we hope to ensure better outcomes for adults living with Dravet syndrome,” explained Professor Perucca.

While more evidence is needed to confirm whether Fintepla reduces the risk of sudden unexplained death in epilepsy (SUDEP), early findings are encouraging. The therapy substantially reduced generalised tonic–clonic seizures (GTCs), the leading risk factor for SUDEP.

Selina Clifford, General Manager ANZ at UCB, said “We are committed to improving the lives of people living with rare and severe forms of epilepsy. As our understanding of the genetic mutations underlying Dravet syndrome continues to grow, we remain focused on developing innovative solutions to address the unique challenges of this complex neurological condition.

“Ensuring Australians have access to the care and treatment they need is a priority, and we welcome the reimbursement of Fintepla as a significant step toward providing new treatment options and support for this vulnerable community.”

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