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Home » NICE Recommends Elosulfase Alfa for Morquio A Syndrome
NICE Recommends Elosulfase Alfa for Morquio A Syndrome
March 22, 2022
The UK’s National Institute for Health and Care Excellence (NICE) has recommended the routine use of BioMarin’s Vimizin (elosulfase alfa) by the National Health Service (NHS) for patients with a rare skeletal disorder, Morquio A syndrome.
The costly once-a-week intravenous infusion replaces the enzyme lacking in people with the syndrome. The enzyme is needed to break down large sugar molecules that the body’s cells are unable to use.
NICE previously rejected the drug in November 2021 because of its high price. Vimizin costs more than $1,000 for each 5-milligram vial. The agency did not disclose the new price that was negotiated with the company for NHS use.
NICE estimates that 90 NHS patients will gain routine access to the product.
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