NICE:Vimzin for routine NHS use in MPS 4A patients - 0 views
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pharmacybiz on 23 Mar 22The National Institute for Health and Care Excellence (NICE) has recommended elosulfase alfa for routine use in the NHS for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome). Elosulfase alfa, also called Vimizin and made by BioMarin, becomes the first disease modifying treatment recommended by NICE for routine NHS use for people with this rare, severely life-limiting condition. NICE's final draft guidance follows the collection of 'real-world' data from 69 people who received NHS-funded treatment since 2015 as part of a managed access agreement. Clinical trial evidence and data collected as part of the managed access agreement, along with expert clinical opinion, shows some long-term benefits with elosulfase alfa treatment which suggest it slows progression of MPS 4A.