FDA OKs Nitisinone for Ultra-Rare Black Urine Disease

FDA OKs Nitisinone for Ultra-Rare Black Urine Disease

(MedPage Today) -- The FDA approved nitisinone (Harliku) for the reduction of urine homogentisic acid (HGA) in adults with alkaptonuria, Cycle Pharmaceuticals announced on Thursday. Alkaptonuria is an ultra-rare hereditary disorder of tyrosine...

Cycle Pharmaceuticals’ HARLIKU™ (nitisinone) Tablets Receive First FDA-Approval as Treatment for Alkaptonuria (AKU)

BOSTON & CAMBRIDGE, England--(BUSINESS WIRE)--Jun 19, 2025-

Nitisinone Becomes First and Only FDA-Approved Treatment for HGA in Adults With AKU

Its approval was based on data from a 3-year trial that enrolled patients with alkaptonuria.

FDA Approves Nitisinone for Treatment of Alkaptonuria

Alkaptonuria affects approximately 1 in 250,000 to 1 million people in the United States.