BioSante's leukemia vaccine gets orphan drug status

BioSante Pharmaceuticals Inc.'s treatment for acute myeloid leukemia received orphan-drug designation from the Food and Drug Administration on March 23 as the vaccine led to increased remission rates in a Phase II study.

In a paper published in the peer-reviewed journal Blood, clinical investigators, led by researchers at Johns Hopkins University, reported on the results of a Phase II study of GVAX AML.

In a study of 54 subjects, those treated with the GVAX AML treatment saw relapse-free and overall survival rates improve by about 30 percent compared with those who didn't receive the treatment.

It's the second BioSante drug candidate to receive the orphan-drug designation in recent weeks.

So-called orphan diseases affect fewer than 200,000 Americans. There are about 7,000 such maladies, most of them serious, that have few or no drugs to treat them.

Getting an orphan-drug designation opens the door to incentives once the Food and Drug Administration approves a medicine for sale in the United States, including seven years' marketing exclusivity and tax breaks.

BioSante acquired a portfolio of cancer vaccines in October through its purchase of Cell Genesys.