YM BioSciences’ highly selective JAK1/2 inhibitor, CYT387 has been granted Orphan Drug Designation. The drug is being developed for the potential treatment of myelofibrosis, a chronic debilitating disease in which a patient's bone marrow is replaced by scar tissue, and for which treatment options are limited.

The designation provides a seven-year period of U.S. marketing exclusivity upon approval as well as the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

"CYT387 is a potent, oral JAK1/2 inhibitor that has been demonstrating very favorable biological activity data in our ongoing Phase I/II trial in myelofibrosis and the granting of Orphan Drug Designation is a key regulatory milestone for our CYT387 clinical development program," said Dr. Nick Glover, chief operating officer of YM BioSciences. "This event provides us with further support for our expanding commitment to this promising drug."

The compound is currently being investigated in a Phase I/II study in myelofibrosis patients at Mayo Clinic, Rochester, NY. The company intends to expand the program to as many as 120 patients at six centers in the U.S., Canada and Australia.