Access to ruxolitinib (Jakavi®) has been facilitated by inclusion in the list of orphan medicines, which are not commercially viable in Australia but treat rare diseases. Ruxolitinib is approved to reduce splenomegaly and disease associated symptoms from inflammatory cytokines in myelofibrosis and the precursor disease, polycythaemia vera. It inhibits cellular Janus kinase pathways to reduce overproduction of abnormal, immature blood cells.

The dose should be titrated, initially based on platelet count up to a maximum of 25mg twice daily, according to safety and efficacy. Dose reduction should be considered when administered in conjunction with cytochrome P3A4 inhibitors, renal impairment, hepatic impairment, anaemia and thrombocytopenia. The degree of survival advantage is still to be confirmed and adverse events are very common.

Consideration for the inclusion of ruxolitinib in the Pharmaceutical Benefits Scheme was deferred in July, pending refinement of its place in clinical practice, access, and price.


  1. Jakavi (ruxolitinib) Australian approved product information. North Ryde: Novartis Pharmaceuticals Australia Pty Limited. Approved 03 July 2013, amended 10 September 2014.
  2. NPS MedicineWise. New Drugs: Ruxolitinib. Aust Prescr 2013: Oct 4.

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