The National Institute for Health and Care Excellence (NICE) has recommended Chiesi’s Lamzede®▼ (velmanase alfa) in their final evaluation document as an option for treating the non-neurological signs and symptoms of mild to moderate alpha-mannosidosis1. The treatment must be started in people under 18 years of age, and can be continued in people who turn 18 while on treatment, in line with a commercial agreement1.
Velmanase alfa is the first and only enzyme replacement therapy recommended by NICE for alpha-mannosidosis and is given to patients once-a-week by intravenous infusion1. It is designed to supplement or replace an enzyme that is missing in patients with alpha-mannosidosis2.
Alpha-mannosidosis is an extremely rare, genetic condition – for example, it is thought to affect around 25 people in England, with many of these children and young people.1 People with the condition have a shortage of the enzyme alpha-mannosidase, which is needed to break down certain sugars in the body2. When this enzyme is missing or does not work properly, these sugars build up inside the cells and may lead to varying levels of signs and symptoms including skeletal abnormalities, hearing loss, intellectual disability, and dysfunction of the immune system1. People with alpha-mannosidosis may experience increasing problems as their disease progresses which require a high level of care, with more severe cases associated with poor survival rates1.
Dr Kamran Iqbal, Head of Medical Affairs, Global Rare Diseases, Chiesi UK&I said: “We are pleased that eligible patients in England and Wales will now be able to access velmanase alfa on the NHS for the first time. The discovery and availability of new treatment options is crucial for those affected by this progressive disease, and we have worked tirelessly over several years with key stakeholders including NICE, patient groups and clinicians to address uncertainties that are common in rare diseases and ensure access for those in need. We are immensely grateful for the hard work and commitment of all involved.”
Bob Stevens, Group Chief Executive, MPS Society said: “On behalf of our alpha-mannosidosis community, I am pleased at the news that NICE has made the decision to make velmanase alfa available to suitable patients in our community in England and Wales. This marks a real change for this community because now they have the possibility of treatment and this is another example of innovative science benefitting our rare patients.”
1 National Institute for Health and Care Excellence. Velmanase alfa for treating alpha-mannosidosis. Final Draft Guidance. Available at https://www.nice.org.uk/guidance/indevelopment/gid-hst10010/documents. Last accessed November 2023.
2 Electronic Medicines Compendium. Lamzede 10 mg powder for solution for infusion. Summary of Medicines Products Characteristics. Available at https://www.medicines.org.uk/emc/product/12836/smpc#about-medicine. Last accessed November 2023.