British pharmaceutical company GSK (LON:GSK) has received a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for its blood-cancer drug momelotinib. This endorsement is a critical step toward potential marketing authorization by the European Commission, which is anticipated in early 2024.
The CHMP's backing is based on encouraging data from clinical trials, including the MOMENTUM phase III study and a subset of patients from the SIMPLIFY-1 phase III trial. These studies evaluated momelotinib's safety and efficacy in treating key symptoms of myelofibrosis, a rare blood cancer characterized by an enlarged spleen, progressive anaemia, and debilitating symptoms.
Momelotinib is designed to inhibit JAK1, JAK2, and ACVR1 signalling pathways, which may improve constitutional symptoms and reduce spleen size while potentially enhancing anaemia benefits. This dual action places momelotinib as a potential first-in-class treatment option for myelofibrosis patients with moderate to severe anaemia, addressing both splenomegaly and disease-related symptoms.
The MOMENTUM trial specifically targeted symptomatic, anaemic myelofibrosis patients who had previously been treated with an approved JAK inhibitor. The study met all primary and key secondary endpoints, showing significant improvements in symptoms, spleen size response, and transfusion independence compared to danazol.
SIMPLIFY-1 compared momelotinib's effectiveness against ruxolitinib in patients who had not previously received JAK inhibitor therapy. The trial's results were focused on a subgroup of patients who presented with anaemia at baseline.
Despite the positive outcomes, common adverse reactions observed during the trials included diarrhoea, thrombocytopaenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain, and cough.
If the European Commission grants approval following this recommendation, momelotinib will be marketed under the proposed trade name Omjjara. This follows its approval in September 2023 by the U.S. Food and Drug Administration (FDA) under the brand name Ojjaara for treating intermediate or high-risk myelofibrosis in adults with anaemia.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.