Pharmacy Prior Authorization Criteria

Nulibry (fosdenopterin)

Indications for Prior Authorization

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.

Criteria

Nulibry

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Both of the following:
• Diagnosis of molybdenum cofactor deficiency (MoCD) Type A
• Genetic mutation in the MOCS1 gene
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

Nulibry

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
• Patient continues to benefit from medication

P & T Revisions

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

References

1. Nulibry Prescribing Information. Origin Biosciences, Inc. Boston, MA. March 2021.
2. 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.
3. Per clinical consultation with pediatrician, April 30, 2021.
4. 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

---

End Notes

1. 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]
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]

---

Revision History

• 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

---