Posted by Michael Wonder on 27 Apr 2018
FDA grants breakthrough therapy designation to Omeros’ MASP-2 inhibitor OMS721 for the treatment of high-risk haematopoietic stem cell transplant-associated thrombotic microangiopathy
26 April 2018 - Discussions on-going with FDA and European regulators for expedited approval.
Omeros Corporation today announced that the U.S. FDA has granted breakthrough therapy designation to OMS721 for the treatment of patients with high-risk haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patients who have persistent TMA despite modification of immunosuppressive therapy.
Breakthrough therapy designation was granted based on data from Omeros’ Phase 2 clinical trial evaluating OMS721 in patients with high-risk HSCT-TMA. To be eligible for enrolment in the clinical trial, HSCT-TMA patients are required to be adults with post-transplant TMA persisting for at least two weeks following immunosuppressive regimen modification (conservative treatment) or more than 30 days post-transplant. This population was chosen to represent a population at risk for poor outcomes, including mortality. These patients often have serious, life threatening co-existing conditions, and mortality rates have been reported to be as high as 100%.
