BioSante Pharmaceuticals, Inc. today announced the receipt of Orphan Drug designation for GVAX CML Vaccine in the treatment of chronic myeloid leukemia (CML) from the FDA’s Office of Orphan Products Development. Orphan drug designation, as granted by the U.S. Orphan Drug Act, is for a product to treat a rare disease or condition that affects fewer than 200,000 people in the U.S. Orphan drug designation qualifies the sponsor of the product for tax credits and seven years of marketing exclusivity, among other benefits.
The orphan drug designation for the GVAX CML Vaccine resulted from BioSante’s third GVAX regulatory submission since acquiring this portfolio of cancer vaccines in October 2009. This is the third orphan drug designation for BioSante; the company also has received orphan drug designations for its vaccines to treat pancreatic cancer and acute myeloid leukemia.
“We continue to develop our vaccine portfolio in cooperation with the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center,” said Stephen M. Simes, president and chief executive officer of BioSante. “Clinical trials of GVAX cancer vaccines are being conducted to treat leukemia, pancreatic cancer and breast cancer, among other cancer types. In addition, we recently announced reinitiation of the GVAX Prostate cancer vaccine program.”
As previously reported, in a clinical study, the GVAX CML vaccine was administered to 19 CML patients with measurable cancer cells, despite taking Gleevec(R) for at least one year (range of 13-53 months). Each patient was given a series of four vaccines administered in three-week intervals while remaining on a stable dose of Gleevec. After a median of 72 months of follow-up, the number of remaining cancer cells declined in 13 patients, eight of whom had increasing disease burden before vaccination. Twelve patients reached their lowest levels of residual cancer cells to date following vaccination. Seven CML patients had complete remission. Patients receiving the GVAX CML vaccine experienced relatively few side effects that included injection site pain and swelling, occasional muscle aches and mild fevers.
The study was conducted by researchers at the Johns Hopkins Kimmel Cancer Center in Baltimore, Maryland, led by Hyam Levitsky, M.D., professor of oncology, medicine and urology at the Cancer Center. The research was funded by the National Institutes of Health.
