The US Food and Drug Administration (FDA) has designated ilixadencel a Regenerative Medicine Advanced Therapy (RMAT) as a treatment for metastatic renal cell carcinoma (RCC). This designation is similar to the FDA’s Breakthrough Therapy Designation, and recognises the therapeutic potential of ilixadencel as well as the unmet patient need for viable therapies for this difficult-to-treat disease. The RMAT designation allows direct guidance from the FDA for the development of ilixadencel.
The RMAT decision was based on the results from the phase 2 MERECA study evaluating the safety and efficacy of ilixadencel in combination with sunitinib in newly diagnosed patients with metastatic RCC. Eighty-six patients were enrolled in this study, 56 of whom received ilixadencel plus sunitinib and 30 of whom received sunitinib alone. Overall, the rate of survival was 54% in the ilixadencel group compared with 37% in the sunitinib group. Median overall survival data remain immature. Objective response rate with ilixadencel plus sunitinib was 42.2% versus 24.0% for sunitinib alone.