Nulibry Approved to Treat Rare Genetic Disorder

February 28, 2021 – The U.S. FDA has approved NulibryTM (fosdenopterin), manufactured by BridgeBio Pharma, to reduce the risk of death due to molybdenum cofactor deficiency (MoCD) Type A.

MoCD is a rare genetic disorder that affects enzymatic activity in the body. In MoCD Type A, this leads to a toxic buildup of S-sulfocysteine (SSC) and other substances that would otherwise be rendered harmless through normal metabolic activity. Onset of symptoms can occur within days of birth, with patients experiencing feeding difficulties, seizures that do not respond to treatment, and irreversible neurological damage.

In clinical studies, Nulibry delivered an 84% three-year survival rate compared to a 55% survival rate for untreated disease. It also reduced SSC urine concentrations, a result sustained for more than 48 months with long-term treatment. Nulibry helps to replace missing molybdenum cofactor, which the body needs to safely break down SSC. It is the first FDA-approved therapy specifically for MoCD Type A, which was previously managed primarily through supportive care and treatment of disease complications.

Recommended dosing is based on the patient’s age and weight. For patients under one year of age, the dose is increased over the course of three months from an initial dose to a maintenance dose. Nulibry is administered once daily via intravenous injection. While it is intended for administration by a healthcare provider, it can be administered at home by a properly trained caregiver if the healthcare provider deems this appropriate.

Nulibry has launched at a wholesale acquisition cost (WAC) of $1,369.86 per 9.5mg vial.