Actelion’s Zavesca (miglustat), the only licensed treatment for Niemann-Pick type C (NP-C) disease, has been approved in the UK and the Republic of Ireland.
NP-C is a rare genetic disease with significant neurological deterioration that can be fatal and affects infants, children and adults. There is currently no cure for the disease, and patient management was previously restricted to symptom relief.
Neurological deterioration is the key feature of NP-C, which manifests in a variety of symptoms including eye movement disorders, balance disorders, difficulty in swallow
ing, slurred and irregular speech, seizures and a lack of muscle control.
Ed Wraith MD, Royal Manchester Children’s Hospital, commented: “For the first time we have an approved therapy for NP-C. The data on the effects of treatment with Zavesca obtained in a clinical trial and in a retrospective cohort study consistently showed a favourable clinical response. As a treating physician I am acutely aware of the importance of reducing progression of neurological symptoms.”
Jean-Paul Clozel, Chief Executive Officer of Actelion, added: "I am very proud that Actelion – together with the scientific community – has been able to demonstrate the role of Zavesca in reducing the progression of clinically relevant neurological symptoms in patients with NP-C. I would like to thank both the patients and their families who, over the years, have been involved in our clinical programme with so much dedication, as well as all the clinical experts for their continuous support.”
Zavesca, which was granted orphan drug status allowing for a faster approval process, is now approved in all EU countries for the treatment of patients with NP-C.