NULIBRY (fosdenopterin)

NULIBRY (fosdenopterin)

Medical Administration / Home Health Administration – intravenous

Diagnosis considered for coverage:

• Indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A

Coverage Criteria:

For diagnosis of Molybdenum Cofactor Deficiency (MoCD) Type A:

• Dose does not exceed 0.9 mg/kg once daily, AND
• Prescribed by or in consultation with a physician who specializes in the treatment of inherited metabolic disorders, AND
• Submission of documentation (e.g., chart notes, laboratory values) confirming 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

Reauthorization Criteria:

• Prescribed by or in consultation with a physician who specializes in the treatment of inherited metabolic disorders, AND
• Patient continues to benefit from medication

Coverage Duration:

• Initial: 1 year
• Reauthorization: 1 year

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information:

• Potential for photosensitivity-avoid or minimize patient exposure to direct sunlight and artificial UV light exposure (i.e., UVA or UVB phototherapy) and adopt precautionary measures (e.g., have the patient wear protective clothing and hats, use broad-spectrum sunscreen with a high sun protection factor (SPF) in patients 6 months of age and older, and wear sunglasses when exposed to the sun). If photosensitivity occurs, advise caregivers/patients to seek medical attention immediately and consider a dermatological evaluation.

Policy Updates:

• 6/15/2021 – New policy approved by P&T.

References:

• Nulibry Prescribing Information. Origin Biosciences, Inc. Boston, MA. March 2021.
• 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.
• Per clinical consultation with pediatrician, April 30, 2021.
• 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