FDA nod for Cycle Pharma’s Harliku for alkaptonuria

Privately-held UK firm Cycle Pharmaceuticals announced that the US Food and Drug Administration (FDA) has approved Harliku (nitisinone) tablets for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU).

Expected  to launch in July 2025, Cycle noted that Harliku will be the first and only FDA-approved treatment for AKU, an ultra-rare genetic metabolic disorder in which patients have a buildup of HGA that leads to osteoarthritis, ochronosis, and complications in the kidneys, and heart. Patients with AKU often develop pain, reduced joint mobility, and require large joint replacements; the symptoms impede their physical functionality, emotional well-being, and quality of life.

The FDA in 2017 approved Cycle’s nitisinone under the trade name Nityr for the treatment of hereditary tyrosinemia type 1 (HT-1).