Fosdenopterin (Nulibry™)

Restricted Product(s)

• fosdenopterin (Nulibry™) intravenous infusion for administration by a healthcare professional 

FDA Approved Use

• For reduction of mortality risk in patients with molybdenum cofactor deficiency (MoCD) Type A

Criteria for Medical Necessity

The restricted product(s) may be considered medically necessary when the following criteria are met:

Initial Criteria for Approval

1. The patient has a diagnosis of molybdenum cofactor deficiency (MoCD) Type A [medical record documentation required]; AND 
2. The diagnosis has been confirmed by genetic testing demonstrating MOCS1 gene mutation [medical record documentation required]; OR 
3. In neonates (i.e., up to 28 days after birth), the diagnosis has been confirmed by both of the following [medical record documentation required]: 
   1. At least one of the following clinical signs and symptoms consistent with the diagnosis: 
      1. Seizures; OR 
      2. Exaggerated startle response; OR 
      3. High-pitched cry; OR 
      4. Axial hypotonia; OR 
      5. Limb hypertonia; OR 
      6. Feeding difficulties; AND 
   2. At least one of the following biochemical markers consistent with the diagnosis: 
      1. Elevated urinary sulfite and/or S-sulfocysteine (SSC); OR 
      2. Elevated xanthine in urine or blood; OR 
      3. Low or absent uric acid in the urine or blood; AND 
4. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND 
5. For requests for injection or infusion administration of the requested medication in an inpatient or outpatient hospital setting, Site of Care Criteria applies (outlined below)*
   
   Duration of Approval: 180 days (6 months)

Continuation Criteria for Approval

1. The patient was approved through Blue Cross NC initial criteria for approval; OR 
2. The patient would have met initial criteria for approval at the time they started therapy [medical record documentation required]; AND 
3. The diagnosis has been confirmed by genetic testing demonstrating MOCS1 gene mutation [medical record documentation required]; AND 
4. The patient has demonstrated a positive clinical and/or biochemical response while using the medication, as demonstrated by at least ONE of the following [medical record documentation required]: 
   1. Decrease in seizure activity; OR 
   2. Improvement in alertness and/or responsiveness; OR 
   3. Improvement in feeding; OR 
   4. Decrease in urinary sulfite and/or S-sulfocysteine (SSC); OR 
   5. Decrease in xanthine levels in urine or blood; OR f. Increase in uric acid levels in urine or blood; AND 
5. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND 
6. For requests for injection or infusion administration of the requested medication in an inpatient or outpatient hospital setting, Site of Care Criteria applies (outlined below)*
   
   Duration of Approval: 365 days (1 year)

*Site of Care Medical Necessity Criteria

1. For requests for injection or infusion administration in an inpatient setting, the injection or infusion may be given if the above medical necessity criteria are met AND the inpatient admission is NOT for the sole purpose of administering the injection or infusion; OR 
2. For requests for injection or infusion administration in an outpatient hospital setting, the injection or infusion may be given if the above medical necessity criteria are met AND ONE of the following must be met: 
   1. History of mild adverse events that have not been successfully managed through mild pre-medication (e.g., diphenhydramine, acetaminophen, steroids, fluids, etc.); OR 
   2. Inability to physically and cognitively adhere to the treatment schedule and regimen complexity; OR 
   3. New to therapy, defined as initial injection or infusion OR less than 3 months since initial injection or infusion; OR 
   4. Re-initiation of therapy, defined as ONE of the following: 
      1. First injection or infusion after 6 months of no injections or infusions for drugs with an approved dosing interval less than 6 months duration; OR 
      2. First injection or infusion after at least a 1-month gap in therapy outside of the approved dosing interval for drugs requiring every 6 months dosing duration; OR 
   5. Requirement of a change in the requested restricted product formulation; AND
3. . If the Site of Care Medical Necessity Criteria in #1 or #2 above are not met, the injection or infusion will be administered in a home-based infusion or physician office setting with or without supervision by a certified healthcare professional.

References

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information

October 2021: Criteria change: Added Site of Care medical necessity criteria. Policy notification given 8/2/2021 for effective date 10/1/2021.
June 2021: Criteria change: Added maximum units; medical policy formatting change. Policy notification given 4/16/2021 for effective date 6/16/2021.
*Further historical criteria changes and updates available upon request from Medical Policy and/or Corporate Pharmacy.