Myelofibrosis is an uncommon type of bone marrow cancer that disrupts your body’s normal production of blood cells and causes extensive scarring in your bone marrow, leading to severe weakness.
A third of the patients have anemia up front, and most patients will develop it throughout their disease. For patients with severe anemia, survival is shortened to about two years. Even with mild anemia, the median survival is 4.9 years. A drug to treat anemia in these patients has been urgently needed.
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Pioneering MF drugs developed over the last decade, such as ruxolitinib, inhibit the harmful JAK signaling, whereas momelotinib is the first drug that inhibits both JAK and ACVR1.
Is The Magic Pill Ready For Myelofibrosis?
To know more about this, researchers analyzed momelotinib against a second medication, danazol, in symptomatic and anemic MF patients previously treated with standard-of-care JAK inhibitor therapy. It enrolled 195 MF patients in 21 countries.
Of the enrolled participants, 130 received momelotinib and 65 danazol. Participants did not know until after 24 weeks which drug they received, and those in the danazol group were allowed to cross over to momelotinib at that time.
Significant improvements in symptoms, spleen size, and anemia measures were observed. Favorable safety and a trend toward improved overall survival were documented.
Participants required fewer transfusions to replace red blood cells and evidenced better oxygen-carrying hemoglobin levels. The study enrolled participants entirely during the COVID time frame, which was remarkable.
Momelotinib met all primary and secondary endpoints and within the short span of six months, there was a trend toward overall survival benefit, which is also remarkable. It can be used as a single pill in the treatment of myelofibrosis in the future.
Source: Medindia
