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NICE approves life-changing medication for treatment of alpha mannosidosis

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The MPS Society is delighted to announce that after five years velmanase alfa (Lamzede®) has been recommended by NICE (The National Institute for Health and Care Excellence) for treating alpha mannosidosis – if started in people under 18 years in England and Wales.

Velmanase alfa, manufactured by pharmaceutical company Chiesi, is the first disease-modifying treatment for people with alpha mannosidosis.

During the five-year evaluation period, the company had to restrict the population to focus on people who start treatment under 18 years and presented data for this population at the last committee (people who turn 18 while on treatment can continue). Whilst we are saddened this recommendation does not include all people the treatment is licensed for, we hope with better disease awareness, all diagnosed eligible people will benefit going forward.

Whilst we are saddened this recommendation does not include all people the treatment is licensed for, we hope with better disease awareness, all diagnosed eligible people will benefit going forward

Alpha mannosidosis is an extremely rare, multi-systemic disorder, which affects multiple organs and is progressive in nature.

Clinical trials of velmanase alfa (Lamzede®) have shown it significantly halts disease progression over a prolonged period offering patients stability, enhanced life expectancy and a life with minimal health-related burden. The NICE re-evaluation looked at clinical trial evidence and feedback from patient and clinical experts.

The committee acknowledged that alpha-mannosidosis is an ultra-rare, serious and debilitating condition that severely affects the lives of people with the condition, and their families and carers. The committee acknowledged the serious and substantial effect on the quality of life of people with the condition and their care giver and that the clinical evidence suggested that velmanase alfa may provide clinical benefit despite the high uncertainty due to the nature and extent of the evidence available.

Bob Stevens, Group Chief Executive of the MPS Society, said:

Today after five long years we celebrate the news that NICE has recommended velmanase alfa for routine use for people with alpha mannosidosis if started before the age of 18 years. It has been a long and at times difficult journey and whilst not all people will have access currently, we hope over time with improved disease awareness all newly diagnosed people will benefit.

The evaluation committee has prepared final draft guidance (FDG) on velmanase alfa for treating alpha-mannosidosis and submitted it to NICE.


Notes for editors

Contact details

For further information or to arrange interviews, please contact the Communications team on 0345 389 9901 or email communications@mpssociety.org.uk.

About MPS Society

Founded in 1982, by Christine Lavery MBE, the Society for Mucopolysaccharide Diseases, or MPS Society, is the only registered charity providing professional support to individuals and families affected by the rare, life-limiting genetic diseases: MPS, Fabry and related conditions in the UK. The MPS Society is transforming the lives of those affected and supports over 1,750 people per year.

About Chiesi Group

Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.

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