CHMP Shares Positive Opinion of Momelotinib for Myelofibrosis/Anemia

The positive opinion marks one of the final steps leading to the agent's potential approval. If approved, momelotinib would be the only agent in Europe available for treating moderate to severe anemia in newly diagnosed and previously treated myelofibrosis, as well as potentially resolving splenomegaly and other symptoms.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has expressed a positive opinion on momelotinib (Ojjaara) as a treatment for patients with moderate to severe anemia with primary myelofibrosis, post–polycythemia vera myelofibrosis, or post-essential thrombocythemia, as well as disease-related splenomegaly according to a press release from GSK.¹

Patients who are JAK inhibitor naïve or have previous treatment with ruxolitinib (Jakafi) can also receive treatment with momelotinib.

The positive opinion marks one of the final steps leading to the agent's potential approval. If approved, momelotinib would be the only agent in Europe available for treating moderate to severe anemia in newly diagnosed and previously treated myelofibrosis, as well as potentially resolving splenomegaly and other symptoms.

“Momelotinib has a differentiated mechanism of action that may address the significant medical needs of [patients with] myelofibrosis, especially those with moderate to severe anemia,” Nina Mojas, senior vice president of Oncology Global Product Strategy at GSK, said in the press release. “The vast majority of [patients with] myelofibrosis will develop anemia, causing them to require transfusions and leading a notable proportion to discontinue treatment. This positive CHMP opinion is a significant step in bringing momelotinib to patients in the EU with this difficult-to-treat blood cancer.”

The positive opinion was supported by several clinical trials, including the phase 3 MOMENTEUM study (NCT04173494) and a patient subgroup from the phase 3 SIMPLIFY-1 study (NCT02101268) with moderate to severe anemia.^2,3

Findings from the MOMENTEUM trial, which included 195 patients, showed a 24-week total symptom score response rate of 24.6% (95% CI, 17.49%-32.94%) among those treated with momelotinib compared with 9.2% (95% CI, 3.46%-19.02%) in those treated with danazol (P =

.0095). Additionally, a splenic volume reduction of 25% was observed in 40.0% (95% CI, 31.51%-48.95%) vs 6.2% (95% CI, 1.70%-15.01%), respectively, as well as a 35% reduction in 23.1% (95% CI, 16.14%-31.28%) vs 3.1% (95% CI, 0.37%-10.68%; P = .0006).

Additionally, in the SIMPLIFY-1 study, which included 432 patients, investigators reported a reduction in total symptom score of 50% or more in 28.4% of patients treated with momelotinib and 42.2% in those treated with ruxolitinib (P = .98).

Common adverse effects across both studies included diarrhea, thrombocytopenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain, and cough.

The FDA approved momelotinib for patients with myelofibrosis and anemia in September 2023.⁴

References

1. GSK receives positive CHMP opinion recommending momelotinib for myelofibrosis patients with anaemia. News release. GSK. November 13, 2023. Accessed November 13, 2023. https://bit.ly/3MEYpOl
2. Mesa RA, Gerds AT, Vannucchi A, et al. MPN-478 MOMENTUM: phase 3 randomized study of momelotinib (MMB) versus danazol (DAN) in symptomatic and anemic myelofibrosis (MF) patients previously treated with a JAK inhibitor. J Clin Oncol. 2022;40(suppl 16):7002. doi:10.1200/JCO.2022.40.16_suppl.7002
3. Mesa RA, Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: a phase III randomized trial of momelotinib versus ruxolitinib in janus kinase inhibitor-naïve patients with myelofibrosis. J Clin Oncol. 2017;34(suppl 34):3844-3850. doi:10.1200/JCO.2017.73.4418
4. Ojjaara (momelotinib) approved in the US as the first and only treatment indicated for myelofibrosis patients with anaemia. News release. GSK. September 15, 2023. Accessed November 13, 2023. https://bit.ly/46eFscj