NovelMed announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).

NM5072 is being developed for PNH patients in the United States and globally. The FDA grants ODD status to new medication intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

PNH is a rare, chronic, inflammatory, and hemolytic disease that occurs when PNH Red Blood Cells (RBCs) breakdown within and outside the blood vessel, causing anemia, which can be fatal. In addition to PNH RBCs, other cells, including Neutrophils, Monocytes, and Platelets, also drive cellular destruction and inflammation, leading to long-term effects on the patient’s health. If the disease remains untreated or partially treated, patients will continue to have anemia and inflammation, potentially leading to chronic pain, fatigue, and other common symptoms of PNH.

Dr. Rekha Bansal, Chief Executive Officer said, “PNH is a rare disease which involves a range of blood cells that contribute to debilitating symptoms for patients, including anemia, fatigue, and severe pain with shorter life span if remains untreated. The current standard of care requires further improvements. We are hopeful that NM5072, with its unique mechanism of action that targets the top of the complement cascade, could become a promising treatment to improve outcomes in these patients.” NM5072 is under regulatory review for multiple indications in the USA and globally.

“We are thrilled by the FDA’s decision, which highlights the demand for groundbreaking therapies for individuals grappling with PNH,” comments Robert Bard, VP Regulatory Affairs. This Orphan Drug Designation approval represents a remarkable stride in therapeutic innovation, showcasing NM5072’s distinct capability to selectively inhibit the alternative pathway (AP) while safeguarding the classical pathway essential for combating infections in PNH patients. NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States.