In draft guidance produced by its Highly Specialised Technologies programme, NICE has asked BioMarin to provide more information about its drug elosulfase alfa (Vimizim) for treating the very rare inherited disease mucopolysaccharidosis type IVa (also known as MPS IVa and Morquio A syndrome). 

The draft guidance asks the company to provide a number of further details to support NICE’s evaluation, including an explanation of the cost of elosulfase alfa, additional economic analyses, information on the budget impact of the drug, and details of the criteria for using elosulflase alfa in clinical practice.  

MPS IVa is a lysosomal storage disease that affects the major organ systems in the body. People born with the disease lack an enzyme - N‑acetylgalactosamine‑6‑sulfatase - that breaks down large sugar molecules (glycosaminoglycans) the body’s cells can’t use. This leads to an accumulation of glycosaminoglycans in the cells of several tissues and organs, causing progressive tissue damage.

For more details, go to: http://www.nice.org.uk/news/press-and-media/nice-asks-company-for-further-information-on-drug-for-rare-inherited-disease-in-draft-guidance