FDA Grants Priority Review to Treatment for Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy
Narsoplimab is an investigational, fully human IgG4 monoclonal antibody was designed to bind to mannan-binding lectin-associated serine protease-2
The FDA has granted priority review to the biologics license application (BLA) for narsoplimab (OMS721) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), according to an announcement from Omeros Corporation.1 Under the Prescription Drug User Fee Act, the regulatory agency must make a decision on the BLA by July 17, 2021. Notably, the FDA does not plan to hold an advisory committee meeting to discuss the application, according to the agency’s filing letter.
“The filing of our BLA by the FDA marks an important milestone on the path to commercialization of narsoplimab,” Gregory A. Demopulos, MD, chairman and chief executive officer of Omeros, stated in a press release. “There is no FDA-approved product for the treatment of transplant-associated TMA, a frequently fatal complication of stem cell transplantation. We appreciate FDA’s collaborative approach throughout the development of our breakthrough therapy-designated product narsoplimab, and we are committed to working closely with the FDA review team to make the drug available to patients who need it.”
The investigational, fully human IgG4 monoclonal antibody was designed to bind to mannan-binding lectin-associated serine protease-2 (MASP-2).2 The lectin pathway is mostly activated by either tissue damage or microbial infection. Notably, unlike other complement inhibitors, narsoplimab has not been found to interfere with the classical complement pathway, which plays a key role with regard to acquired immune response to infection. The investigational antibody was developed to prevent complement-mediated inflammation and endothelial damage without affecting other innate immunity pathways.