USANew treatments and approvals
First approval for alkaptonuria in the USA: nitisinone reduces homogentisic acid in urine
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The FDA approved nitisinone to reduce homogentisic acid in the urine of adults with alkaptonuria. The approval is based on a three-year trial with 40 patients. In those on the drug, this acid fell by 88% over one year. In those not taking the drug, it increased.
What does it mean for patients and families?
The approval is based on the reduction of the acid in urine. Its effect on joint and heart complaints is not the subject of this approval. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Alkaptonuria (AKU)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.