Outcomes of consultation document leans towards a NO for treatment of Alpha Mannosidosis

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Outcomes of consultation document leans towards a NO for treatment of Alpha Mannosidosis

NICE, the drug decision making body, publicly released their interim decision on the 23 May 2018 not to recommend velmanase alfa, the treatment for individuals aged 6 years and over suffering from Alpha Mannosidosis.

Members of the public have until the 13 June 2018 to submit their views and feedback to NICE. The evaluation committee will then reconsider the evidence and their decision at a further meeting.

Meeting minutes and committee papers can be found here.

The drug received licensing approval from the European Medicines Agency (EMA) in March 2018. The licensed drug velmanase alfa, is one of the only treatments for patients over 6 years which addresses the underlying cause of the disease. For younger children there is an option of having a haematopoietic stem cell transplantation (HSCT).

Alpha Mannosidosis is an ultra-rare multi-system disease, which affects multiple organs and is progressive in nature.

Clinical trials have shown reduction in disease progression, improved health and quality of life in most patients.

Although the evaluation committee acknowledged that Alpha Mannosidosis was an exceptionally rare condition they were unable to give a positive recommendation due to limitations in the clinical evidence presented and the uncertainty in clinical efficacy and outcomes from the clinical trial evidence. The committee also felt that there still remained uncertainties within the economic model.

Children and adults who were currently enrolled on a clinical trial continue to receive compassionate access in the short term.

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 patients over 6 years old 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.”

The MPS Society will continue to work with patients, clinicians, Chiesi pharmaceuticals and NICE to put forward the patient views and to try and influence the decisions of NICE in favour of recommending this treatment for all patients diagnosed with Alpha Mannosidosis.

About the MPS Society

The Society for Mucopolysaccharide Diseases (MPS Society) is the only registered UK charity providing professional support to over 1300 families in the UK affected by MPS and related disease. The MPS Society provides a pioneering advocacy service to individual families as well as funding innovative and life changing clinical and academic research.

As a registered charity the Society is entirely supported by voluntary donations and fundraising.


Sophie Thomas
Advocacy Support Team Manager
MPS Society
Telephone: 0345 389 9901

Download a Guide to Alpha Mannosidosis