Orphan drug designation for Sapacitabine

[Birgitta-A]

Hi all,
Now the new drug Sapacitabine has been granted orphan drug designation for the treatment of both AML and MDS.
http://production.investis.com/cyc/i...10/2010-07-01/
Kind regards
Birgitta-A

[Neil Cuadra]

Just last month Cyclacel Pharmaceuticals, the manufacturer of Sapacitabine, announced the results from a Phase II study. Sapacitabine was found to help MDS patients who failed to improve with hypomethylating agents: azacitidine, trade name Vidaza, and decitabine, trade name Dacogen.

Sapacitabine works by damaging the DNA of cancer cells, preventing the cells from multiplying. For the technical minded, it's a nucleoside analogue that interferes with the DNA polymerase enzyme.

Phase II studies assess how well a drug works with different does. For Sapacitabine, a Phase III study in a larger patient population comes next. Phase III studies measure how effective a drug is compared with the treatment patients would otherwise get. MDS patients who have failed Vidaza and Dacogen currently have no good options, so this brings new hope for further effective treatment.