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Condition update published on

NICE leans towards a no for the treatment of Alpha Mannosidosis


NICE (National Institute of Clinical Excellence) the drug decision making body have publicly released their interim decision not to recommend Velmanase alfa (Lamzede®), for treating patients with non-neurological signs and symptoms of mild to moderate alpha mannosidosis. Although NICE have acknowledged the seriousness of the condition and its impact on patients' and families' quality of life, the committee had uncertainties over the clinical benefit to patients and with the company’s economic model. This is not NICE’s final decision and the committee is proposing to meet again on the 18 January 2023.

Members of the public have until the 25 November 2022 to submit their views and feedback to NICE. The evaluation committee will then reconsider the evidence and their decision at the proposed meeting in January 2023.

The committee are interested in receiving comments on the following:

  • Has all of the relevant evidence been taken into account?

  • Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

  • Are the provisional recommendations sound and a suitable basis for guidance to the NHS?

The link for submitting comments is:

Individuals with alpha mannosidosis have been waiting for a decision following the drug's licensing by the European Medicines Agency in 2018.

Velmanase alfa (Lamzede®), is the only treatment for alpha mannosidosis which addresses the underlying cause of the disease in patients with mild to moderate disease.

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 to significantly halt disease progression over a prolonged period of time offering patients stability, enhanced life expectancy and a life with minimal health related burden.

Bob Stevens, Group Chief Executive of the Society for Mucopolysaccharide Diseases said:

"We are disappointed by the announcement from NICE not to recommend treatment for this small group of patients”

"Velmanase alfa would offer eligible patients some hope that the long-term disease burden, mortality and morbidity, would be greatly reduced and disease progression delayed to enable them to have a good quality of life”

“In contrast The Scottish Medicines Consortium (SMC) accepted velmanase alfa (Lamzede®) for patients with alpha mannosidosis within its ultra-orphan pathway in September 2022”.

The MPS Society will continue to work with patients, clinicians, Chiesi Limited, NHSE and NICE to put forward the patient views and to try and influence the decisions of NICE in favor of recommending this treatment for all eligible patients.

About the Patient organisation

MPS Society


Media enquiries:

Sophie Thomas (Senior Head of Patient Services and Clinical Liaisons):

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