Hematology Nephrology

Accelerated approval for OMS721 in HSCT-TMA

After receiving Breakthrough Therapy designation for OMS721 in HSCT-TMA, the company just announced that they are in discussion with the FDA for an accelerated approval in this indication and therefore will soon be initiating a rolling BLA submission.

The update comes after Omeros had reported very encouraging date with the complement inhibitor in 19 stem cell TMA patients where the estimated median survival was an order of magnitude greater than that seen in historical data.

Thrombotic microangiopathy occurs in 30% of patients undergoing hematopoietic stem cell transplantation.
Various previous studies have already found that complement inhibition with Eculizumab can significantly increase the chance of survival.

Mortality rates in patients which develop a severe form of the disease are reported to be up to 80%. Given that a substantial fraction of the HSCT patients are children, an expedited path to approval makes a lot of sense for this promising new drug, especially given the good side effects profile of OMS721.