The U.S. Food and Drug Administration (FDA) approved today NULIBRY™ (fosdenopterin), a treatment developed by BridgeBio and its affiliate Origin Biosciences for molybdenum cofactor deficiency (MoCD) Type A. MOCD Type A is an extremely rare metabolic disorder that affects less than 150 patients around the world; the FDA approval is the first for any treatments in reducing the mortality risk for patients. Neil Kumar, Ph.D., BridgeBio founder and Chief Executive Officer, stated, “The FDA’s approval of NULIBRY means that patients with MoCD Type A and their families have an approved therapy for the first time. It also reflects our belief that every life matters and that no disease is too rare to address. As is often true in rare disease drug development, this was a community effort in which we were able to play a part – we’d like to thank the patients, caregivers, physicians, scientists, and advocates who played an essential role in achieving this important milestone.”
Read more here.