Posted by Michael Wonder on 25 Feb 2021
NICE draft guidance recommends sapropterin for children with rare inherited metabolic condition phenylketonuria
25 February 2021 - NICE has today issued draft guidance for public consultation which recommends sapropterin (Kuvan, BioMarin) for treating phenylketonuria in children aged up to 18.
There were limitations with the economic analyses presented, which made it difficult to estimate the cost effectiveness of sapropterin. However, when accounting for the benefits and cost of the treatment the committee concluded that sapropterin in children is likely to be within the range NICE considers an effective use of NHS resources.
