NICE recommends Vimizin (elosulfase alfa) for MPS IVA- BioMarin Pharma
In final draft guidance as part of its Highly Specialised Technologies Programme, NICE has provisionally recommended Vimizin (elosulfase alfa), from BioMarin Pharma, for the treatment of a very rare inherited lysosomal storage disease MPS IVA when used as part of a managed access agreement.
Elosulfase alfa is an enzyme replacement therapy consisting of the N-acetylgalactosamine-6-sulfatase enzyme. It replaces the enzyme lacking in people with MPS IVa. Before elosulfase alfa became available there was no treatment for the underlying disease. Because the condition is so rare and the symptoms so diverse, there is no standard treatment or pathway of care.
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