BOSTON — Cycle Pharmaceuticals has announced the U.S. launch of HARLIKU™ (nitisinone) Tablets, marking the first FDA-approved treatment for alkaptonuria (AKU), a rare genetic metabolic disorder also known as Black Bone Disease.
AKU affects an estimated one in 1,000,000 individuals in the United States. The condition is caused by a buildup of homogentisic acid (HGA) in the body, which can lead to joint pain, stiffness, and tissue discoloration over time.
HARLIKU works by inhibiting an upstream enzyme responsible for producing HGA, reducing its levels in urine by up to 97%, according to the company. Clinical studies showed that patients treated with nitisinone experienced improvements in pain, energy, and physical function.
“With the launch of HARLIKU, Cycle Vita can now provide patients with AKU the individualized support they need to navigate their treatment from diagnosis onwards,” said Jamie Ray, Director of the Patient Support Program at Cycle Pharmaceuticals. The company’s support platform, Cycle Vita™, offers personalized assistance throughout the treatment journey.
Nick Sireau, CEO and Chair of Trustees of the AKU Society, welcomed the milestone. “The launch of HARLIKU marks an exciting milestone for our AKU community,” he said. “As the first commercially available treatment for AKU in the United States, it will empower patients to take a more proactive role in their care, paving the way for personalized treatment discussions and helping to reduce barriers to access.”
