multiple myeloma
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The US Food and Drug Administration (FDA) has granted orphan drug designation for tasquinimod as a treatment for multiple myeloma (MM).
Tasquinimod is an immunomodulatory, anti-metastatic, and anti-angiogenic compound being developed by Active Biotech AB.
The company says tasquinimod works by inhibiting the function of S100A9, a pro-inflammatory protein that is elevated in MM and other malignancies.
S100A9 is believed to aid cancer development by recruiting and activating immune cells such as myeloid-derived suppressor cells.
Active Biotech AB says that, by targeting the S100A9 pathway, tasquinimod interferes with the accumulation and activation of myeloid-derived suppressor cells in the tumor microenvironment, which decreases immune suppression and angiogenesis.
Tasquinimod also inhibits the hypoxic response in the tumor by binding to HDAC4, according to Active Biotech AB.
The company says tasquinimod has produced “robust results” in animal models of MM, and research presented at the AACR Annual Meeting 2015 supports this statement.
Investigators found that tasquinimod reduced tumor growth and improved survival in mouse models of MM. And these effects were associated with reduced angiogenesis in the bone marrow.
Tasquinimod was previously under development as a treatment for prostate cancer, but research suggested the drug did not have a favorable risk-benefit ratio in this patient population.
About orphan designation
The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.
Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.
