CurrentMass General Brigham Health PlanPolicy N/A

Nulibry (fosdenopterin) coverage for molybdenum cofactor deficiency (MoCD) Type A

Defines prior authorization, coverage criteria, and authorization durations for Nulibry (fosdenopterin) under Mass General Brigham Health Plan pharmacy benefit for members with MoCD Type A.

Policy Summary

PayerMass General Brigham Health Plan

PolicyNulibry (fosdenopterin) coverage for molybdenum cofactor deficiency (MoCD) Type A

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateNov 1, 2021

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation of diagnosis (clinical or genetic confirmation) and, for reauthorization, evidence of therapeutic response.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

3Initial approval duration

12Reauthorization duration (months)

MoCD Type AIndication covered

Medical Necessity Criteria for Nulibry (fosdenopterin)

Initial Authorization — Medical Necessity Criteria

Authorization may be granted when ALL of the following criteria are met and documentation is provided:

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ALL of the following

Diagnosis documentation (ONE of)
- Documented diagnosis of molybdenum cofactor deficiency Type A
- Diagnosis of molybdenum cofactor deficiency Type A confirmed by genetic testing
- Prenatal genetic diagnosis
Neonatal diagnosis option (for neonates up to 28 days)
- Onset of clinical signs and symptoms consistent with MoCD Type A within the first 28 days after birth (e.g., seizures, feeding difficulties, high-pitched cries, exaggerated startle reactions, increased/decreased muscle tone)
- Onset of laboratory signs and symptoms consistent with MoCD Type A within the first 28 days after birth (e.g., elevated urinary sulfite and/or S-sulphocysteine, elevated xanthine in urine or blood, or low or absent uric acid in the urine or blood)

Continuation / Reauthorization Criteria

Reauthorization will be granted when ALL of the following criteria are met:

ALL of the following

Diagnosis of molybdenum cofactor deficiency Type A confirmed by genetic testing
Documented therapeutic response as evidenced by ONE of the following
The policy lists 'Documented therapeutic response as evidenced by ONE of the following' but does not enumerate the specific response criteria in the provided text.

Authorization and Documentation Requirements

Prior Authorization

Prior authorization required

Prior authorization is required for coverage of Nulibry; authorizations may be reviewed for members new to the plan who are currently receiving treatment (excludes samples or manufacturer patient assistance program supplies).

Documentation Required

Documentation of diagnosis and response

Providers must supply documentation of diagnosis (clinical or genetic confirmation) and, for reauthorization, documentation of therapeutic response; neonatal cases may be documented via clinical or laboratory signs within the first 28 days after birth.

Initial authorization: clinical diagnosis of MoCD Type A, genetic confirmation of MoCD Type A, or prenatal genetic diagnosis.
Neonates (up to 28 days): clinical signs (e.g., seizures, feeding difficulties, high-pitched cry, exaggerated startle, abnormal muscle tone) or laboratory signs (e.g., elevated urinary sulfite/S-sulphocysteine, elevated xanthine, or low/absent uric acid) within first 28 days.
Reauthorization: documented therapeutic response as evidenced by clinical documentation and genetic confirmation of MoCD Type A.

Coverage Scope, Durations, and References

Policy Scope and Status

Scope summaryDefines prior authorization, coverage criteria, and authorization durations for Nulibry (fosdenopterin) under Mass General Brigham Health Plan pharmacy benefit for members with MoCD Type A.

Coverage stancecovered_with_criteria

Policy statusCURRENT

Key Policy Durations and Indication

Initial approval duration3 months

Reauthorization duration12 months

Indication coveredMoCD Type A

Limits: Initial approvals will be granted for 3 months.

Reauthorizations will be granted for 12 months.

References:

• Nulibry [package insert], Origin Biosciences, Inc.; February 2021. (package insert)

• Schwahn BC et al. Lancet 2015; 386:1955-1963.

• Atwal PS, Scaglia F. Mol Genet Metab. 2016;117(1):1-4.

• ClinicalTrials.gov studies of ORG0001 referenced.