The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved the use of GSK’s momelotinib (Omjjara) for moderate to severe anaemia in adult patients with myelofibrosis.
The decision marks a significant development in the treatment of a rare blood cancer that disrupts the production of blood cells due to scar tissue in the bone marrow.
Momelotinib is an oral tablet administered once a day. It targets janus-associated kinases JAK1 and JAK2, which regulate cytokines involved in inflammation, blood component production and immune system regulation.
It also hinders activin A receptor type 1, reducing the over-production of hepcidin, a protein that causes anaemia by trapping iron in the liver.
The approval of momelotinib is underpinned by data from a Phase III clinical trial involving 195 patients who had previously received a JAK inhibitor.
Participants were given 200mg of the medication or 300mg of an alternative myelofibrosis treatment, danazol, over a 24-week period.
Momelotinib significantly outperformed danazol in reducing symptoms and spleen size.
25% of subjects in the momelotinib arm experienced at least a 50% reduction in symptoms versus 9% on danazol.
22% saw spleen volume decrease by at least 35% versus 2% on danazol.
Infections, reduced blood platelet count, dizziness, headache, cough, diarrhoea, nausea, stomach-ache and fatigue are common side effects linked to momelotinib.
MHRA Healthcare Quality and Access interim executive director Julian Beach stated: “Myelofibrosis patients often rely on blood transfusions to counter their anaemia, but studies have shown that these transfusions are often linked to reduced quality of life and survival.
“Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.
“We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.”
Approximately 40% of myelofibrosis patients are diagnosed with moderate to severe anaemia, and nearly all develop it over time, necessitating increased care and in many cases blood transfusions.
