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Southampton Health Technology Assessments CentreNews

SHTAC is critically appraising the company’s submission to NICE on elosulfase alfa for treating mucopolysaccharidosis type IVA (Morquio A syndrome)

Published: 8 December 2014

SHTAC is assessing the clinical-effectiveness and cost-effectiveness of elosulfase alfa for treating mucopolysaccharidosis type IVA (Morquio A syndrome), for the National Institute for Health and Care Excellence (NICE). This will inform an appraisal for NICE’s new Highly Specialised Technology (HST) evaluations process, which considers new drugs for very rare conditions.

Morquio A syndrome is one of a group of inherited metabolic disorders called mucopolysaccharidoses (MPSs). The MPSs are caused by a lack of lysosomal enzymes, which are needed to break down sugar in the body. People with the condition have multiple physical difficulties, including short stature, spinal deformities, abnormalities in cartilage and bone development, large unstable knees, elbows and wrists, enlargement of the spleen and liver and clouding of the cornea in the eye. As a result of these disabilities, people may develop neck instability or dislocation, gradual loss of the ability to walk, breathing problems and respiratory infections. These events tend to reduce the life expectancy of people with this condition. This is a rare disease, with only about 100 people in the UK known to have the disorder.

Morquio A syndrome is currently incurable so the management of the condition has concentrated on treating symptoms. Enzyme replacement therapy with the drug elosulfase alfa (Vimizim) is one new treatment currently being tested in patients with Morquio A.

The new Highly Specialised Technology (HST) programme enables NICE to make recommendations on the use of very high cost drugs for people with rare conditions for the NHS in England. The programme continues the work previously done by the Advisory Group for National Specialised Services (AGNSS). NICE is expected to issue guidance on elosulfase alfa to the NHS in October 2015.

For more information on SHTAC’s research metabolic conditions please visit our Research page.

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