Momelotinib is now available on the PBS for patients with myelofibrosis who have moderate to severe anaemia.
The Pharmaceutical Benefits Advisory Committee (PBAC) recommended the listing after determining the JAK inhibitor demonstrated non-inferior effectiveness and safety compared to ruxolitinib, with specific benefits for anaemia management.
From 1 April 2025, haematologists can prescribe momelotinib (Omjjara) for patients with intermediate or high-risk primary myelofibrosis, post-polycythaemia vera myelofibrosis, or post-essential thrombocythaemia myelofibrosis.
The PBS listing applies to patients who are either JAKi naïve or have previously been treated with ruxolitinib. All eligible patients must have a haemoglobin level below 100 g per litre prior to commencing treatment.
In its November 2024 meeting, the PBAC accepted GlaxoSmithKline’s claim that momelotinib provides a “clinically meaningful improvement in anaemia-related outcomes” and offers a “safety advantage in lowering the risk of anaemia adverse events” compared to ruxolitinib.
The listing requires Authority Required approval, with initial treatment applications processed through telephone/online systems. Patients must have confirmed diagnosis through bone marrow biopsy, with risk status classified according to recognised prognostic scoring systems.
PBAC also advised that the equi-effective doses were momelotinib at a mean daily dose of 186.2 mg compared to ruxolitinib at a mean daily dose of 26.2 mg.
The committee noted flow-on changes to ruxolitinib’s listing that will allow switching between the two agents under limited circumstances, providing greater flexibility for haematologists managing this complex condition.
Momelotinib is available in 100 mg, 150 mg, and 200 mg tablet formulations. The PBS listing specifies patients may qualify for PBS-subsidised treatment under this restriction twice in a lifetime, with an exemption for patients reinitiating treatment following pregnancy.
Aussie-led study provides real-world guide to ruxolitinib use
Meanwhile, research led by Australian haematologists offers practical strategies for managing cytopenias and infections in myelofibrosis patients treated with ruxolitinib.
The case-based review, published in eJHaem [link here], presents seven common clinical scenarios to guide clinicians through the complexities of JAK inhibitor therapy.
Lead author and Monash University researcher Dr Liesl Butler, alongside colleagues from Alfred Health, Central Coast Haematology, and the UK’s Guy’s and St Thomas’ NHS Foundation Trust, shared recommendations based on 15 years of experience using ruxolitinib and other JAK inhibitors.
“The treatment landscape in MF is evolving rapidly, however ruxolitinib remains the standard of care and has been established as a safe and effective compound in clinical trials, pooled analyses, expanded-access studies and post marketing surveillance,” they wrote.
“Haematological adverse events are expected with JAK inhibition and can be managed with dose modification, trans-fusion, or a combination of the two.”
The study outlined several crucial management approaches for common complications. For anaemia, the researchers emphasised that baseline anaemia should not prevent ruxolitinib initiation, with numerous analyses showing “improvement in symptoms, spleen volume and survival in those with pre-existing anaemia and transfusion dependence”.
When managing thrombocytopenia, the authors recommended a case-by-case approach for patients with platelets below 50 × 10⁹/L, considering low-dose ruxolitinib if appropriate.
Infection risk was highlighted as a significant concern, with researchers recommending screening for various infectious diseases before commencing therapy and considering prophylactic treatments based on individual risk.
The paper also addressed the challenging issue of non-melanomatous skin cancers, noting that rates “might be higher in continents with high lifetime UV exposure, such as Australia and Africa”.
For patients developing disease progression or blast phase, the authors outlined various strategies including alternative JAK inhibitors and combination therapies.
The research was partly funded by Australia’s Medical Research Future Fund and the Leukaemia Foundation of Australia.
“A range of therapies are being explored in the context of ruxolitinib failure, whilst some of the novel agents offer potential for disease modification,” the authors concluded.