Insider Insights

UK: “First of its kind” MAA leads to NICE approval


The National Institute for Health and Care Excellence (NICE) on 21st March 2022 published final draft guidance recommending the routine use in the national health service (NHS) of BioMarin’s Vimizin (elosulfase alfa) for the treatment of mucopolysaccharidosis type 4A. The draft guidance follows the collection of real-world data since 2015 as part of a managed access agreement, the “first of its kind to have been attempted in the NHS in England”, according to Helen Knight, acting interim director of medicines evaluation at NICE.  

NICE indicates that around 100 children a year will be eligible for the drug.  

The draft final guidance reverses previous guidance issued in November 2021 rejecting the drug for routine use in the NHS. Notably, NICE in both instances expressed criticisms of the submission noting that the company’s analyses were very uncertain. Indeed in its final draft guidance, the committee notes that “the company used the same limited economic model structure for the review as for the original guidance. This was despite encouragement to improve the structure to better reflect the characteristics of MPS 4A, target population and treatment benefits of elosulfase alfa”. 


  1., “NICE recommends elosulfase alfa for routine NHS use for patients with rare life-limiting metabolic disorder”, 21st March 2022 
  2., “Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]”, 21st March 2022 

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