The FDA has granted orphan drug designation to nintedanib for the treatment of mesothelioma.
Nintedanib is an oral triple angiokinase inhibitor that simultaneously inhibits vascular endothelial growth factor receptors (VEGFR 1-3), platelet-derived growth factor receptors, and fibroblast growth factor receptors (FGFR 1-3) signaling pathways. These three different angiokinase receptors, which are not yet targeted by any currently available therapies, play an important role not only in angiogenesis but also in tumor growth and the development of metastases.
The designation was granted based in part on data from the phase II cohort of the ongoing phase II/III LUME-Meso trial, an international, multicenter, randomized, double-blind, placebo-controlled trial. It evaluated the efficacy and safety profile of nintedanib plus chemotherapy (pemetrexed/cisplatin) followed by nintedanib monotherapy, versus placebo plus chemotherapy (pemetrexed/cisplatin) followed by placebo monotherapy, in patients with histologically confirmed, unresectable malignant pleural mesothelioma.
The phase III LUME-Meso trial (NCT01907100) is currently recruiting patients worldwide. The phase III portion of the trial plans to randomize 450 patients at 137 sites in 27 countries.