New treatment option available for Dravet syndrome

Global biopharmaceutical company UCB said the UK’s National Institute for Healthcare Excellence (NICE) issued a positive final assessment decision (FAD) for its FINTEPLA (fenfluramine) oral solution.

FINTEPLA is used for the treatment of seizures associated with Dravet syndrome as an add-on treatment to other antiepileptic medicines in patients aged two years and over.

Dravet syndrome is a rare, lifelong epilepsy that begins in early childhood.

Its incidence rate is estimated at one in 15,700 people. Common problems associated with Dravet syndrome include: prolonged and frequent seizures, behavioral and developmental delays, movement and balance problems, orthopedic problems , delayed language and speech problems, growth and nutrition problems, sleep disorders, chronic infections and sensory integration disorders.

Patients with Dravet syndrome face a 15-20% mortality rate due to SUDEP (Sudden Unexpected Death in Epilepsy), prolonged seizures, and seizure-related accidents such as drowning and infections.

“Because Dravet syndrome is particularly difficult to treat, we are acutely aware of the unmet medical need for people living with this condition. This news underscores the wider recognition of addressing this unmet need and echoes UCB’s continued commitment to the epilepsy community,” said Claire Brading, Managing Director, UK and Ireland, UCB . “We are delighted to have received this positive decision from NICE and look forward to making this treatment option more widely available as a complementary therapy for patients with Dravet syndrome in the UK.”

Galia Wilson, chair and trustee of Dravet Syndrome UK, whose son has the condition, said: “Dravet Syndrome is a catastrophic disease that has a devastating impact on all aspects of life. People with the disease and their families urgently need improved treatment and care. »

Wilson said Dravet syndrome can be unpredictable, like most current treatments, fenfluramine may not work for everyone. However, she added, from clinical and real-world experience, it can be transformative.

New processing option

Professor Helen Cross, Prince of Wales chair on childhood epilepsy and head of UCL-ICH’s neuroscience unit, said one of the many complexities of Dravet syndrome is that the seizures are usually resistant to treatment.

“Children with Dravet syndrome experience frequent and prolonged seizures, affecting their quality of life and that of their caregivers,” Cross said. “Fenfluramine has demonstrated impressive seizure reduction in clinical studies, so this positive decision by NICE signifies a new, effective treatment option and hope for improved quality of life for affected families.”

Other companies such as the Austrian biotech a:head bio AG are also working on the fight against neurological disorders, including Dravet syndrome. Last year, the company secured more funding to recreate human brain disorders in a lab environment.

At the end of 2022, a:head is planning a Series A funding round to prepare for its next phase of growth.

Cover image: Shutterstock

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