Iclusig

Iclusig is the brand name for ponatinib, an oral, multi-target tyrosine kinase inhibitor used in the treatment of certain Philadelphia chromosome–positive leukemias. Ponatinib inhibits the BCR-ABL tyrosine kinase and several other kinases, including PDGFR, VEGFR, FGFR, KIT, and RET, and retains activity against leukemic cells that harbor the T315I mutation, which confers resistance to many other TKIs.

Iclusig is indicated for adults with chronic myeloid leukemia (CML) or Philadelphia chromosome–positive acute lymphoblastic leukemia (ALL) that is resistant or intolerant to prior TKI therapy, or for disease characterized by the T315I mutation. Regional labeling may vary; in the United States, FDA approval for these indications was granted in 2012. Availability and dosing guidelines may differ by country.

Administration and dosing typically involve oral once-daily dosing. The initial dose is commonly 45 mg once daily, with subsequent dose reductions to lower levels (such as 30 mg or 15 mg daily) based on efficacy, tolerability, and safety concerns. Dosing may be adjusted to balance therapeutic benefit with the risk of adverse events, and monitoring for response and toxicity is required.

Safety and risks: Iclusig carries boxed warnings for arterial and venous thrombotic events and for hepatotoxicity, including liver failure. Other major risks include pancreatitis, wound healing impairment, hypertension, and other cardiovascular and hepatic adverse events. Common side effects include rash, abdominal pain, nausea, fatigue, headache, and elevated liver enzymes. Treatment requires careful patient selection, risk–benefit assessment, and regular monitoring.

History and availability: Ponatinib (Iclusig) was developed for resistant Philadelphia chromosome–positive leukemias and is marketed by Takeda in many regions. It remains an important therapy for patients with limited alternatives, with dosing and monitoring guided by regulatory labeling.