FDA Grants Tasquinimod Orphan Drug Status to Treat Multiple Myeloma

The small molecule immunomodulatory drug tasquinimod has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma, according to Active Biotech.

Tasquinimod has been developed primarily for the treatment of prostate cancer, but its mechanism of action suggests that it could be used to treat other cancers, having shown robust activity in multiple myeloma animal models.

The FDA’s orphan drug status is meant to accelerate the development and approval of drugs designed to treat, diagnose, or prevent rare diseases — those affecting fewer than 200,000 people in the U.S. The designation provides monetary and other incentives, and a seven-year marketing exclusivity period against competition.

Despite the development of new myeloma treatments that greatly improved the prognosis and survival of patients, the disease is highly heterogeneous and patients often become resistant to approved drugs. Multiple myeloma patients thus face an unmet medical need.

Tasquinimod is a small molecule that affects the tumor’s ability to grow and spread to distant parts of the body through distinct mechanisms of action.

First, tasquinimod disrupts the tumor immunosuppressive environment by acting on myeloid-derived suppressor cells (MDSCs), a type of immune cell that helps tumors progress by preventing the recruitment and activation of tumor-killing immune cells.

MDSCs are also involved in the formation of new tumor blood vessels and in the metastatic process, and preclinical studies have also demonstrated that tasquinimod can disrupt the formation of tumor blood vessels, reducing the oxygen and nutrient supply, and decrease the ability of cancer cells to spread to distant areas of the body.

Immunosupressive cells like MDSCs play a key role in myeloma progression because they promote immune evasion, blood vessel formation, and metastasis. This makes tasquinimod a promising candidate for the treatment of these patients.

Prior clinical studies of tasquinimod in metastatic castration-resistant prostate cancer patients (mCRPC) have shown a good safety profile and a significant prolongation in the time to disease progression or death. But tasquinimod has failed to induce overall survival benefit in a randomized Phase 3 trial.

The company is now testing tasquinimod in combination with other prostate cancer agents. Active Biotech is also seeking a partner for the continued development of tasquinimod in multiple myeloma.