Pharmacy Prior Authorization Criteria

Nulibry (fosdenopterin)

For diagnosis of Molybdenum cofactor deficiency (MoCD) Type A
Indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

AND

Patient has clinical and/or laboratory signs and symptoms consistent with MOCD Type A (e.g., seizures, limb/axial hypertonia, elevated levels of urinary sulfite/SSC [s-sulfocysteine] or xanthine in blood/urine, low uric acid in blood/urine)

AND

Prescribed by or in consultation with a physician who specializes in the treatment of inherited metabolic disorders

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

Prescribed by or in consultation with a physician who specializes in the treatment of inherited metabolic disorders

AND

2025-04-02, 2024-04-02, 2023-03-13, 2022-06-08, 2022-03-29, 2021-05-06

Nulibry Prescribing Information. Origin Biosciences, Inc. Boston, MA. October 2022.
Study of ORGN001 (formerly ALXN1101) in neonates, infants and children with molybdenum cofactor deficiency (MOCD) type A. ClinicalTrials.gov identifier: NCT02629393. Updated February 26, 2021. Accessed April 12, 2021. https://www.clinicaltrials.gov/ct2/show/study/NCT02629393.
Mechler, K., Mountford, W., Hoffmann, G. et al. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency. Genet Med 17, 965–970 (2015). https://doi.org/10.1038/gim.2015.12

In patients with a presumptive diagnosis of MoCD Type A, confirm the diagnosis of MoCD Type A immediately after initiation of Nulibry treatment. In such patients, discontinue Nulibry if the MoCD Type A diagnosis is not confirmed by genetic testing. [1,2]
Primary and secondary outcome measures observed in the pivotal trial were overall survival, Gross Motor Function Measure, number of patients who can feed orally, head circumference measurement, length measurement, weight measurement, and BMI measurement. [2]

2025-04-02: Annual Review
2024-04-02: Annual Review
2023-03-13: Annual review - no criteria changes
2022-06-08: Removed submission of medical records and/or paid claims requirement from prior authorization criteria
2022-03-29: Annual Review - No criteria changes
2021-05-06: New Program