Regulatory Affairs

Omeros’ OMS721 gets FDA breakthrough therapy designation for HSCT-TMA

PBR Staff Writer Published 27 April 2018

Omeros’ MASP-2 inhibitor OMS721 has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for the treatment of patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

The breakthrough designation for the drug is especially for HSCT-TMA patients with persistent TMA in spite of modification of immunosuppressive therapy.

OMS721 is a human monoclonal antibody that particularly targets mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

The breakthrough therapy designation makes OMS721 eligible for an FDA priority review while enabling Omeros to roll out submission of portions of its application.

Omeros chairman and CEO Gregory Demopulos said: “High-risk TMA following hematopoietic stem cell transplant carries an extremely high mortality rate, and no treatments are approved for this devastating disorder.

“We appreciate FDA’s recognition of the potential for OMS721 to improve outcomes – most importantly survival – for these patients, and we look forward to working closely with the Agency to accelerate the development and approval of OMS721.”

OMS721’s latest breakthrough therapy designation was given based on the findings of a phase 2 trial which assessed the MASP-2 inhibitor in patients with high-risk HSCT-TMA.

The drug demonstrated an estimated median survival that was an order of magnitude greater than that for a matched historical control.

Omeros said that additional analysis of the data assessed 100-day mortality, a key endpoint previously used as an approval endpoint in HSCT. The company said that this analysis demonstrated that patients treated with OMS721 had improved survival relative to the historical control.

In June 2017, OMS721 was granted a breakthrough therapy designation from the FDA for the treatment of Immunoglobulin A (IgA) nephropathy. It was followed by orphan designation granted by the FDA in August 2017 to the drug for the same indication.

Earlier this year, Omeros said that the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) had given a positive opinion for the company’s application for orphan drug designation for the drug in the treatment of primary Immunoglobulin A nephropathy (IgAN).

Image: FDA Building 51 in Silver Spring, Maryland which houses the Center for Drug Evaluation and Research. Photo: courtesy of The U.S. Food and Drug Administration/