Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) Tablets, marking a pivotal moment as it becomes the first and only FDA-approved treatment for Alkaptonuria (AKU). This ultra-rare genetic metabolic disorder causes a debilitating buildup of homogentisic acid (HGA) in the body, leading to severe osteoarthritis, ochronosis, and serious complications in the kidneys and heart. These symptoms profoundly impact patients' lives, often resulting in chronic pain, limited joint mobility, and the need for extensive joint replacements.

Set to launch in July 2025, HARLIKU's approval is based on a randomized, no-treatment controlled study involving 40 AKU patients. This foundational research, conducted as part of the National Institutes of Health (NIH) intramural program by Dr. Wendy J. Introne and her team, showed that nitisinone significantly improved key patient-reported outcomes such as pain, energy levels, and physical functioning over three years.

While HARLIKU offers immense hope, it's important to be aware of the associated safety information. Potential side effects include ocular symptoms (like keratitis and corneal opacities) and hyperkeratotic skin plaques, both linked to elevated plasma tyrosine levels. The drug may also lead to blood disorders such as leukopenia and thrombocytopenia, necessitating careful monitoring of patients' blood counts. Additionally, HARLIKU can interact with other medications, as it acts as a moderate CYP2C9 inhibitor and a weak CYP2E1 inducer, requiring potential dosage adjustments for co-administered drugs. This approval, however, represents a monumental step forward for the AKU community, providing a long-awaited and much-needed therapeutic option.