Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU.

Launching in July 2025, 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 approval of HARLIKU is based on data from a randomized, no-treatment controlled study of 40 patients with AKU. As part of the intramural research program of the National Human Genome Research Institute at the National Institutes of Health (NIH), Dr. Wendy J. Introne, MD and her team showed that nitisinone helped patients improve pain, energy levels, and physical functioning after three years of treatment, assessed using the 36-Item Short-Form Survey.

Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals commented, "We are deeply grateful for Cycle’s collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH, whose pioneering work laid the foundation for this FDA approval. We look forward to making HARLIKU available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities."

“The approval of HARLIKU is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU,” said Dr. Wendy J. Introne, MD, of NIH’s National Human Genome Research Institute (NHGRI).

Building on the company’s previous success in rare diseases, HARLIKU will be Cycle Pharmaceuticals’ eighth commercial product in the US.