Momelotinib
Treatment of intermediate or high-risk primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis in patients with moderate to severe anaemia who are JAK inhibitor naïve or have been treated with ruxolitinib.
Decision on record
- Meeting Nov 2024 Recommended Myelofibrosis with moderate to severe anaemia
Access path
- Nov 2024Recommended · restricted
vs ruxolitinib
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the listing of momelotinib, on the basis that it should be available as a General Schedule, Authority Required (Telephone/Online) listing for initial treatment and an Authority Required (STREAMLINED) listing for continuing treatment for intermediate or high-risk primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis in patients with moderate to severe anaemia and who are Janus …PSD · Nov 2024
7.2 The PBAC considered there was a moderate need for a new JAK inhibitor in this disease area and momelotinib would provide patients with an alternative to ruxolitinib, with potentially lower incidence of anaemia (see paragraph 6.48) and fewer red blood cell transfusions (see paragraph 6.28).PSD · Nov 2024
6.60 The submission presented a cost-minimisation of momelotinib versus ruxolitinib in patients with myelofibrosis and moderate to severe anaemia, based on the JAK inhibitor naïve population in the SIMPLIFY-1 trial only, given the limitations associated with the clinical evidence for JAK inhibitor experienced patients (primary endpoint for superiority versus best available therapy not met in SIMPLIFY-2, danazol comparator 24PSD · Nov 2024
6.61 The key components of the cost minimisation approach are summarised in Table 11.PSD · Nov 2024
6.52 The submission described momelotinib as non-inferior in terms of effectiveness and overall safety compared with ruxolitinib in JAK inhibitor naïve patients with myelofibrosis and moderate to severe anaemia. Further, the submission claimed that momelotinib provides a clinically meaningful improvement in anaemia-related outcomes and has a safety advantage in lowering the risk of anaemia adverse events compared to ruxolitinib.PSD · Nov 2024
• Momelotinib demonstrated a statistically significant non-inferior splenic response to ruxolitinib, meeting the primary endpoint of the SIMPLIFY-1 trial. The EMA assessment report for momelotinib (November 2023) noted that the selected non-inferiority margin seemed to be arbitrarily chosen, without clinical judgement on the importance of loss of efficacy of momelotinib compared to the reference of ruxolitinib.PSD · Nov 2024
6.2 The PBAC noted and welcomed the input from individuals (16), health care professionals (2) and organisations (3) via the Consumer Comments facility on the PBS website. The comments described the impact of anaemia on quality of life and the impacts this has on family, work and social life, with exhaustion and fatigue limiting patient’s ability to function.PSD · Nov 2024
6.86 A risk sharing arrangement was not proposed in the submission. For more detail on PBAC’s view, see section 7 PBAC outcome.PSD · Nov 2024
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated by design.
The submission presented a cost-minimisation of momelotinib versus ruxolitinib in patients with myelofibrosis and moderate to severe anaemia, based on the JAK inhibitor naïve population in the SIMPLIFY-1 trial only, given the limitations associated with the clinical evidence for JAK inhibitor experienced patients (primary endpoint for superiority versus best available therapy not met in SIMPLIFY-2, danazol comparator PSD · 2024
Decision context
PopulationAdult patients with intermediate or high-risk primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis with moderate to severe anaemia (haemoglobin < 100 g/L) who are JAK inhibitor naïve or previously treated with ruxolitinib.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended · restricted | ruxolitinib | — | RCT · Spleen volume reduction and total symptom score |
Consumer voice
Consumer input described the significant impact of anaemia on quality of life, with exhaustion and fatigue limiting daily functioning across family, work and social life. Consumers highlighted that momelotinib would provide an alternative to ruxolitinib with less anaemia, potentially reducing transfusion requirements and offering an effective option for ruxolitinib-refractory patients.
The comments described the impact of anaemia on quality of life and the impacts this has on family, work and social life, with exhaustion and fatigue limiting patient's ability to function. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to intermediate or high-risk disease; TGA label includes all risk categories without specifying risk stratification.