The European Medicines Agency (EMA) has validated GSK’s marketing authorization application (MAA) for momelotinib to treat myelofibrosis.
With a differentiated mechanism of action, momelotinib has inhibitory ability along three important signalling pathways, Janus kinase (JAK) 1, and JAK2 and activin A receptor type I (ACVR1).
These pathways may address the significant medical needs of myelofibrosis patients with anaemia.
JAK1 and JAK2 inhibition may improve constitutional symptoms and splenomegaly.
The company stated that the regulatory application is based on the data obtained from important Phase III trials that include the pivotal MOMENTUM trial.
The double-blind, randomised, global Phase III clinical trial was designed for evaluating the safety and efficacy of momelotinib to treat and reduce important hallmarks of the disease, including symptoms, blood transfusions (due to anaemia) and splenomegaly (enlarged spleen).
It was conducted in myelofibrosis patients with symptoms and were anaemic and who have previously received treatment with an FDA-approved JAK inhibitor.
GSK stated that the trial met all the primary and key secondary endpoints that include Transfusion Independence (TI) rate, Splenic Response Rate (SRR), and Total Symptom Score (TSS).
It noted that a Committee for Medicinal Products for Human Use (CHMP) regulatory action is expected by the end of next year.
Currently, a New Drug Application for momelotinib is under regulatory review with the US Food and Drug Administration (FDA).
The regulatory body has allotted a Prescription Drug User Fee Act action date of 16 June next year.
The company said that momelotinib is currently not approved in any market.
If approved, it is expected to become the only medicine to address important manifestations of myelofibrosis, such as symptoms, anaemia, and splenomegaly.