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UK patients who tested Kuvan for phenylketonuria will get supplies after trials end, says company

BMJ 2019; 365 doi: https://doi.org/10.1136/bmj.l1874 (Published 24 April 2019) Cite this as: BMJ 2019;365:l1874
  1. Deborah Cohen
  1. London

BioMarin, a US biotech company, has said that all UK patients who have taken part in clinical trials of sapropterin dihydrochloride (Kuvan) for phenylketonuria (PKU) will be able to continue taking the drug after the trials end.

The statement comes after the BBC’s Newsnight highlighted that some patients had not continued to receive the drug once the trials ended.1

This seemed at odds with the Declaration of Helsinki, the global research ethics agreement saying that provisions should be made “for all participants who still need an intervention identified as beneficial in the trial,” although BioMarin insisted, “To the best of our knowledge, BioMarin is treating all known ex-trial patients. If there are any ex-trial patients who are not being treated and would like to be treated, they should talk to their treating physician and apply for treatment.”

Kuvan is currently the only drug licensed to …

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