NICE broadens sapropterin guidance for treating phenylketonuria

pharmafile | August 20, 2021 | News story | Medical Communications  

NICE has extended its approval of sapropterin (also called Kuvan and made by BioMarin) to pregnant women and people aged up to 22 years with rare inherited metabolic condition phenylketonuria.

Following public consultation on NICE’s previous draft guidance which recommended sapropterin for children up to 18 years old, the committee agreed that PKU is a particular concern if poorly controlled during pregnancy because it can cause severe congenital defects in unborn children.

Currently, only pregnant women with PKU who aren’t able to control their condition through diet alone are able to access sapropterin on the NHS. This new recommendation will remove this requirement and could allow women to have sapropterin earlier in their pregnancy with potentially better outcomes for their unborn children.

The committee also acknowledged that sapropterin could prevent long-term irreversible brain damage in children, because childhood is the most critical period for brain development.

There were limitations with the clinical evidence and economic analyses presented, which made it difficult to estimate the cost effectiveness of sapropterin. However, it was concluded that sapropterin in children and in pregnant women is likely to be within the range NICE considers an effective use of NHS resources.

Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Assessment at NICE, said: “Although we’re pleased to now be able to recommend sapropterin for people up to 22 years of age and pregnant women, it’s disappointing not to have been able to extend the recommendation to all adults.

“Even when considering some additional potential benefits of sapropterin in this group, the price of the drug was too high to allow it to be considered an acceptable use of NHS resources. The committee were aware that generic products could be available in the near future and hoped these would be priced to allow access to this drug for all adults with PKU.”

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