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Elosulfase alfa for treating mucopolysaccharidosis type IVA (Morquio A syndrome) - NICE guidance now available

Published: 14 January 2016

The National Institute for Health and Care Excellence (NICE) has issued guidance to the health service on the use the drug elosulfase alfa for treating mucopolysaccharidosis type IVA (Morquio A syndrome), informed by a report produced by SHTAC.

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 such as Morquio A for the NHS in England. NICE’s guidance is that elosulfase alfa (Vimizim) is recommended as a possible treatment for people with Morquio A only if they sign up to the managed patient access agreement in the managed access agreement. The managed access agreement includes rules for starting and stopping treatment with elosulfase alfa, and for assessing how well the treatment is working.

This is the second guidance that NICE has issued as part of the HST programme, with guidance on further treatments forthcoming.

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

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