Harliku FDA Approval History

Last updated by Judith Stewart, BPharm on June 30, 2025.

FDA Approved: Yes (First approved June 19, 2025)
Brand name: Harliku
Generic name: nitisinone
Dosage form: Tablets
Company: Cycle Pharmaceuticals
Treatment for: Alkaptonuria

Harliku (nitisinone) is a hydroxyphenyl-pyruvate dioxygenase inhibitor indicated for the reduction of urine homogentisic acid in adult patients with alkaptonuria.

• Alkaptonuria (AKU) is an ultra-rare genetic metabolic disorder caused by mutations in the HGD gene. It is characterised by an accumulation of homogentisic acid (HGA) that leads to osteoarthritis, ochronosis (a buildup of blue-black pigment in certain tissues), and complications in the kidneys and heart.
• Harliku contains nitisinone, which works in the treatment of AKU by reducing HGA.
• Nitisinone was first approved for the treatment of hereditary tyrosinemia type 1 (HT-1) in 2002, and is available under the brand names Orfadin and Nityr.
• FDA approval of Harliku was supported by results of a randomized, no-treatment controlled study of 40 patients study conducted by the National Human Genome Research Institute at the National Institutes of Health (NIH). Patients treated with Harliku demonstrated improvements in pain, energy levels, and physical functioning after three years of treatment (assessed using the 36-item short-form survey).
• Harliku tablets are administered orally, once daily.
• Warnings and precautions associated with Harliku include ocular symptoms and hyperkeratotic plaques on the soles and palms due to elevated plasma tyrosine levels, and leukopenia and severe thrombocytopenia.
• Common adverse reactions (>1%) include elevated tyrosine levels, keratitis and thrombocytopenia.

Development timeline for Harliku

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer