Posted by Michael Wonder on 20 Aug 2021
NICE draft guidance extends recommendation for sapropterin to pregnant women and people aged up to 22 years with rare inherited metabolic condition phenylketonuria
20 August 2021 - NICE has today issued final draft guidance which now recommends sapropterin (Kuvan; BioMarin) as an option for treating phenylketonuria in pregnant women until they give birth as well as for treating the condition in people until they turn 22.
Following public consultation on NICE’s previous draft guidance which recommended sapropterin for children up to 18 years old, the committee agreed that phenylketonuria is a particular concern if poorly controlled during pregnancy because it can cause severe congenital defects in unborn children.
Currently, only pregnant women with phenylketonuria who aren’t able to control their condition through diet alone are able to access sapropterin on the NHS. This new recommendation could allow women to have sapropterin earlier in their pregnancy with potentially better outcomes for their unborn children.
