Orphan drug status for ruxolitinib


The US Food & Drug Administration has conferred orphan drug status on ruxolitinib for the treatment of the bone marrow disease myelofibrosis.

Approval came on the heels of data from the COMFORT trial. This clinical trial enrolled 528 patients, all of whom were refractory or resistant to existing myelofibrosis therapy or were ineligible for an allogenic bone marrow transplantation. Furthermore, all patients had previously undergone a splenomegaly and were symptomatic.

Ruxolitinib is a twice daily oral inhibitor of the JAK1 and JAK2 enzymes , which become deregulated in myelofibrosis.

In people with myelofibrosis, scar tissue takes over their bone marrow. Blood cell production therefore falls to the liver and the spleen.

Source: HemOncToday

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