La Jolla Pharmaceutical Company has reached an agreement with the European Medicines Agency (EMA) on the design of a pivotal study of LJPC-401, La Jolla’s novel formulation of synthetic hepcidin. The pivotal study will be a randomized, controlled, multi-center study in beta thalassemia patients suffering from iron overload, a major unmet need in an orphan patient population. The primary endpoint will be a clinically relevant measurement directly related to iron overload. La Jolla plans to initiate this pivotal study in mid-2017.
We very much appreciate the EMA’s support of advancing LJPC-401’s development for this major unmet need,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “The EMA’s insights have been invaluable as we sought to design a pivotal study that would best evaluate LJPC-401’s potential to help beta thalassemia patients suffering from iron overload. Based on our recently announced Phase 1 results, and the fact that hepcidin is the body’s natural regulator of iron absorption and distribution, we remain confident that LJPC-401 can help restore normal or near-normal levels of iron in patients suffering from iron overload and its devastating consequences.”