The FDA stated that Fast Track designation was granted for oral azacitidine for myelodysplastic syndromes (MDS) because azacitidine, known in its injectable form as Vidaza, is approved to treat all subtypes of MDS, and because it will potentially provide a safer, more comfortable, more convenient and more efficient route of administration of azacitidine.
Pharmion currently markets Vidaza for the treatment of patients with MDS. Pharmion said that it is exploring oral azacitidine's utility in the treatment of MDS and other cancers where demethylation may provide an anti-tumor effect.
Oral azacitidine is the subject of a Phase I multi-center, open label dose escalation trial that will assess the maximum tolerated dose, dose limiting toxicities and safety of a seven day, multi-cycle oral dosing regimen of oral azacitidine in patients with MDS and AML. In addition, the trial will examine pharmacokinetics and pharmacodynamic effects of orally administered Azacitidine, as compared with parenteral Vidaza.