Dravet syndrome Archives

Global biopharma company UCB says the UK’s National Institute for Healthcare Excellence (NICE) has issued a positive Final Appraisal Determination (FAD) for its FINTEPLA (fenfluramine) oral solution. FINTEPLA is used for the treatment of seizures associated with Dravet syndrome as an add-on therapy to other anti-epileptic medicines for patients two years of age and older. Dravet syndrome is a rare, lifelong epilepsy that begins in infancy. It has an estimated incidence rate of one in 15,700 people. Common issues associated with Dravet syndrome include: prolonged and frequent seizures, behavioral and developmental delays, movement and balance problems, orthopedic conditions, delayed language and speech issues, growth and nutrition issues, sleeping difficulties, 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. “Given that Dravet syndrome is particularly challenging to treat, we are acutely aware of the ongoing unmet medical need for people living with this condition. This news underscores the wider recognition of addressing this unmet need and echoes UCB’s ongoing commitment to the epilepsy community,” said Claire Brading, managing director, UK & Ireland, UCB. “We are delighted to have received this positive NICE decision and look forward to making this treatment option more widely available as an add-on therapy for Dravet syndrome patients in the UK.” Galia Wilson, chair and trustee of Dravet Syndrome UK, whose son has the condition, said: “Dravet syndrome is a catastrophic condition which has a devastating impact on every aspect of life. Individuals with the condition and their families are in urgent need of improved treatments and care.” Wilson said as Dravet syndrome can be unpredictable, like many of the current treatments, fenfluramine may not work for all. However, she added, from clinical and real-life experience, it can be transformative. New treatment option Professor Helen Cross, The Prince of Wales's Chair of Childhood Epilepsy & Head of UCL-ICH Neurosciences Unit, said one of the many complexities of Dravet syndrome is that seizures are usually treatment-resistant. “Children with Dravet syndrome suffer from frequent and prolonged epileptic seizures, affecting quality of life for both them and their caregivers,” Cross said. “Fenfluramine has demonstrated an impressive reduction in seizures in clinical studies, so this positive decision from NICE means an effective new treatment option and hope for an improvement in quality of life for families concerned.” Other companies such as Austrian biotech a:head bio AG are also working on combating neurological disorders including Dravet syndrome. Last year, the company secured more funding to recreate human brain disorders in a lab setting. In late 2022, a:head is planning a series A financing round to prepare for its next growth phase.

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