NICE Extends Recommendation for Sapropterin for Phenylketonuria 

Priscilla Lynch 

August 27, 2021

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance which now recommends sapropterin (Kuvan, made by BioMarin) as an option for treating phenylketonuria (PKU) in pregnant women until they give birth as well as for treating the condition in people until they turn 22 years of age.

Current treatment for the rare, inherited, chronic and life-long metabolic condition is a lifetime adherence to a severely protein-restricted diet, which cuts out most natural proteins, together with dietary supplementation. The aim of the treatment with sapropterin is to reduce blood phenylalanine levels and relax the protein-restricted diet as much as possible in people with PKU.

Only pregnant women with PKU who are not able to control their condition through diet alone are currently able to access sapropterin on the NHS. This new recommendation could allow women to take sapropterin earlier in their pregnancy with potentially better outcomes for their unborn children.

In a further change from the earlier draft guidance, sapropterin is now also recommended for people aged 18 years until they turn 22 years. The committee felt people would benefit if treatment with sapropterin could be continued for as long as possible during final brain development and transition into adulthood. However, sapropterin is only cost-effective in this group if they continue to take it at the dose they were having when they were under 18 years.

There are currently around 2000 people with PKU in NHS care in England.

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.”

This article originally appeared on Univadis, part of the Medscape Professional Network.


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