Boston, Mass.– The U.S. Food and Drug Administration has approved Cycle Pharmaceuticals’ HARLIKU™ (nitisinone) Tablets as the first-ever treatment for alkaptonuria (AKU), a rare and progressive genetic disorder that causes a harmful buildup of homogentisic acid (HGA) in the body.
The approval marks a significant milestone for AKU patients in the United States, who have long faced limited treatment options for a condition that leads to debilitating joint damage, ochronosis (dark pigmentation of connective tissue), and complications involving the heart and kidneys.
HARLIKU, which is expected to launch in July 2025, is approved for the reduction of urine HGA levels in adults with AKU. It becomes the first and only FDA-authorized therapy targeting the root biochemical cause of the disease.
“This is an important advance for the AKU community,” said Dr. Wendy J. Introne of the National Human Genome Research Institute (NHGRI) at the National Institutes of Health. “We’re hopeful this treatment will ease the considerable burden AKU places on patients’ daily lives.”
The FDA’s decision was supported by a randomized, controlled study conducted under the NIH’s intramural research program, involving 40 patients. Over three years, patients treated with nitisinone experienced improvements in pain, energy levels, and physical function, measured using the 36-Item Short-Form Health Survey.
Cycle Pharmaceuticals Chief Strategy Officer Steve Fuller acknowledged the collaboration with NIH researchers, including Dr. Introne and Dr. Bill Gahl, as instrumental to the drug’s development and approval. “We are deeply grateful for the groundbreaking work that made this possible. We’re committed to making HARLIKU available to AKU patients across the U.S. and supporting this underserved community.”
HARLIKU will become Cycle Pharmaceuticals’ eighth commercial product in the U.S., expanding the company’s focus on treatments for ultra-rare and orphan diseases.
