EU CHMP recommends approval of Xermelo (telotristat ethyl) for carcinoid syndrome diarrhea - Lexicon Pharmaceuticals

Lexicon Pharmaceuticals announced that the Committee for Medicinal Products for Human Use, the committee of the European Medicines Agency responsible for human medicines, has adopted a positive opinion for the Marketing Authorization Application filed by Lexicon collaborator Ipsen for Xermelo (telotristat ethyl) 250 mg to treat carcinoid syndrome diarrhea in combination with somatostatin analog therapy in adults inadequately controlled by SSA therapy.

The Xermelo MAA was based on two randomized Phase III studies, TELESTAR and TELECAST. The TELESTAR study was a global, pivotal, double-blind Phase III study for telotristat ethyl in 135 patients from 12 countries with carcinoid syndrome diarrhea whose symptoms were not adequately controlled on SSA therapy. The primary objectives were to evaluate the safety and tolerability of telotristat ethyl and the effectiveness of the drug on the reduction in the average number of daily bowel movements over a 12-week study period. The TELECAST study was designed similarly to the TELESTAR study to provide additional efficacy and safety information in 76 patients with carcinoid syndrome diarrhea. The primary endpoints were the percent change from baseline in u5-HIAA at Week 12 and incidence of treatment emergent adverse events (TEAEs).

Comment: the drug was approved by the FDA on February 28, 2017 for the treatment of carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSA therapy.