YM Biosciences has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to CYT387, its JAK1/2 inhibitor for the treatment of myelofibrosis.
Myelofibrosis is a debilitating condition in which bone marrow is replaced by scar tissue.
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CYT387 is currently being investigated in an oral Phase I/II clinical study involving myelofibrosis patients at Mayo Clinic, New York.
The clinical results showed that CYT387 demonstrated promising evidence of biological activity, including significant spleen size reduction, favourable haematological changes and improvement in constitutional symptoms.
Orphan drug designation is granted to drugs that treat disease conditions affecting fewer than 200,000 patients in the US.
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