CurrentCentenePolicy CP PHAR.471

Fosdenopterin (Nulibry)

Defines Centene's medical necessity criteria, initial and continuation authorization requirements, dosing limits, approval durations, exclusions, coding implications, and prescriber/documentation requirements for fosdenopterin (Nulibry) for treatment of molybdenum cofactor deficiency (MoCD) type A across Commercial, HIM, and Medicaid lines of business.

Policy Summary

PayerCentene

PolicyFosdenopterin (Nulibry)

Policy CodePolicy CP PHAR.471

Change TypeCoverage criteria update (FDA approval and annual reviews)

Effective Date02.26.21

Next Review Date

Key ActionProvider must submit documentation supporting that the member has met all approval criteria, including diagnosis confirmation and current weight.

POLICY UPDATE CHANGES

2Q 2021: Drug is FDA approved; criteria updated per FDA labeling including removal of age restriction, added presumptive neonatal pathway with required genetic confirmation on reauthorization, added prescriber requirement (neonatologist), removed baseline urinary SSC/xanthine/uric acid documentation, added requirement for body weight documentation, increased continued approval duration from 6 to 12 months, and added MoCD type B as not covered.

2Q 2022 - 2Q 2025 annual reviews: no significant clinical changes; references and appendices updated including 2024 consensus guideline updates to Appendix D.

1FDA-approved indication(s)

0.9 mg/kg/dayMaximum daily dose

6m / 1mInitial approval durations (confirmed / presumptive)

Continued approval duration

Coverage Summary

Policy status: covered with criteria for fosdenopterin (Nulibry) for treatment of MoCD type A per Centene criteria. The policy follows FDA labeling: Nulibry is indicated to reduce the risk of mortality in patients with MoCD type A and coverage is limited to MoCD type A when all specified criteria are met (diagnosis confirmation or neonatal presumptive pathway, prescriber specialty, documentation of weight, and dosing limits). The dosing approach includes age-based titration for neonates with a maximum maintenance dose of 0.9 mg/kg/day and specific vial quantity recommendations. The policy explicitly states MoCD type B is not covered under this policy.

Key thresholds and quick facts

FDA‑approved indication(s)Reduce risk of mortality in patients with molybdenum cofactor deficiency (MoCD) type A

Maximum daily dose0.9 mg/kg/day

Initial approval duration (genetically confirmed)6 months

Initial approval duration (presumptive neonate)1 month

Continued approval duration12 months

Presumptive neonatal initial pathway age limit< 28 days old

Initial Therapy Criteria

Initial Approval Duration

ANY of the following

Genetically confirmed diagnosis: approval duration 6 months6 months
Presumptive neonatal diagnosis: Presumptive diagnosis (neonate): approval duration 1 month1 month

Continuation Therapy Criteria

Continued Therapy (A. MoCD type A)

Continued therapy covered when ALL of the following are met:

ALL of the following

Eligibility for continuation
- Currently receiving via Centene: Member currently receiving medication via Centene benefit
- Previously met initial criteria: Member has previously met initial approval criteria
Genetic confirmation for presumptive initial diagnosis: If initial diagnosis was presumptive, diagnosis has been confirmed by genetic testing (presence of MOCS1 mutation) (see Appendix D)

Other Indications & Exclusions

Other Diagnoses/Indications (Non-MoCD type A)

Coverage for other uses only when meeting specified formulary/non-formulary or off-label pathways:

ANY of the following

Formulary/PDL pathway: For drugs on the formulary/PDL: follow no coverage criteria policy for the relevant line of business (CP.CPA.190 commercial; HIM.PA.33 health insurance marketplace; CP.PMN.255 Medicaid)
Non-formulary pathway: For drugs NOT on the formulary/PDL: follow non-formulary policy for the relevant line of business (CP.CPA.190 commercial; HIM.PA.103 marketplace; CP.PMN.16 Medicaid)
Off-label pathway: If requested use is not specifically listed under Diagnoses/Indications NOT authorized and initial criteria do not apply, refer to off-label use policy (CP.CPA.09 commercial; HIM.PA.154 marketplace; CP.PMN.53 Medicaid)

Applicable Codes

HCPCS - FosdenopterinHCPCSCovered

J1809

Injection, fosdenopterin, 0.1 mg

HCPCS - Previously used miscellaneous codes (removed)HCPCSNot Covered

J3490	Unclassified drugs (previously referenced; removed)
C9399	Unclassified drugs or biologicals (previously referenced; removed)

Provider Actions & Authorization Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Prescriptions must be written by or in consultation with a neonatologist, neurologist, or a metabolic specialist (e.g., pediatric geneticist) experienced in the management of inborn errors of metabolism. Prior authorization requests must include the member's diagnosis and current weight (kg).

Affected code: J1809

Documentation Required

Submit Supporting Documentation

Submit supporting documentation with the prior authorization request. Acceptable documentation includes office chart notes, relevant laboratory results, genetic test reports, and any other clinical information that supports the requested use and demonstrates that the member meets the policy criteria.

Clinical Evidence

Key evidence: a prospective cohort study published in Lancet (2015) demonstrated the efficacy and safety of cyclic pyranopterin monophosphate (cPMP) substitution in severe MoCD type A. As context for guideline references, the policy notes updates to Appendix D based on the 2024 consensus guideline revisions included in recent reviews.

Background

Drug description and indication: Fosdenopterin (Nulibry) is a cPMP replacement therapy (cyclic pyranopterin monophosphate) indicated to reduce the risk of mortality in patients with MoCD type A. Dosing uses an age-based titration for neonates (see titration schedule) with a maintenance cap of 0.9 mg/kg/day. The product is supplied as a lyophilized powder in single-dose vials of 9.5 mg for reconstitution.

Policy quick facts

Policy numberCP PHAR.471

SubjectFosdenopterin (Nulibry) coverage policy

PayerCentene

StatusCURRENT

Effective date2021-02-26

Last review date

Definitions

Glossary: cPMP — cyclic pyranopterin monophosphate.

MOCS1 — molybdenum cofactor synthesis 1 gene (genetic confirmation of MoCD type A involves MOCS1 mutation testing).

MoCD — molybdenum cofactor deficiency.

SSC — S-sulfocysteine (biochemical marker referenced for monitoring response).

Revision History

04.13.21 (P&T 05.21)material_change

2Q 2021: Drug FDA approved; policy criteria updated per FDA labeling — removed age restriction; added presumptive neonatal initial-approval pathway (genetic confirmation required on re-authorization); added prescriber requirement (neonatologist and specialists in inborn errors of metabolism); removed requirement for baseline urinary SSC/xanthine/uric acid documentation; added requirement to document current body weight; increased continued approval duration from 6 to 12 months; added MoCD type B as a diagnosis/indication not covered.

02.27.22 (2Q 2022)annual_review

2Q 2022 annual review: no significant clinical changes; references reviewed and updated.

09.28.22 (2Q 2022 template update)annual_review

Policy Summary

PayerCentene

PolicyFosdenopterin (Nulibry)

Policy CodePolicy CP PHAR.471

Change TypeCoverage criteria update (FDA approval and annual reviews)

Effective Date02.26.21

Next Review Date

Key ActionProvider must submit documentation supporting that the member has met all approval criteria, including diagnosis confirmation and current weight.

On This Page

Continuity-of-care

Enrolled in state/product with continuity regs: Member is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC_PHARM.03A and CCPHARM.03B)
Not applicable option retained in policy structure

Evidence of clinical response: Member is responding positively to therapy as evidenced by improvement in clinical outcomes (e.g., improved symptoms, achievement of motor milestones, decreased seizure activity, lack of clinical deterioration)

Evidence of biochemical response: Or member shows positive biochemical outcomes (e.g., decreased or normalized urinary S-sulfocysteine [SSC] or xanthine levels; increased or normalized uric acid levels)

Weight documentation: Documentation of current weight in kilograms

Dose increase constraint: If request is for a dose increase, new dose does not exceed 0.9 mg/kg per day0.9 mg/kg/day

Refer to Appendix E for vial quantity recommendations

Continued Approval Duration

Continued therapy approval duration: 12 months12 months

Documentation Required

Genetic Confirmation Required for Presumptive Initial Approvals

If initial approval was granted based on a presumptive neonatal diagnosis, genetic confirmation is required for re-authorization. Genetic confirmation must demonstrate a pathogenic MOCS1 mutation (see Appendix D).

Billing Rule

Dose and Vial Quantity

Dose must not exceed 0.9 mg/kg per day. Approvals should permit the number of vials necessary to achieve the required dose based on the member's body weight. Refer to Appendix E for vial quantity recommendations.

Affected code: J1809
Maximum dose: 0.9 mg/kg/day
See Appendix E for vial guidance

Documentation Required

Evidence of Response Required for Continuation

For continuation/reenrollment requests, provide documentation demonstrating a positive response to therapy and the member's current weight in kilograms. Evidence of response may include clinical improvement (e.g., improved symptoms, attainment of motor milestones, reduced seizure activity, absence of clinical deterioration) and/or biochemical improvement (e.g., decreased or normalized urinary S-sulfocysteine or xanthine, increased or normalized uric acid).

2Q 2022: template changes to other diagnoses/indications and continued therapy sections; references and policy template updated.

05.23 (2Q 2023)annual_review

2Q 2023 annual review: no significant clinical changes; references reviewed and updated.

01.11.24 (2Q 2024)annual_review

2Q 2024 annual review: no significant clinical changes; references reviewed and updated (Appendix/References updated per routine review).

03.06.25 (P&T 05.25) (2Q 2025)annual_reviewLatest

2Q 2025 annual review: no significant clinical changes; Appendix D clinical and laboratory signs/symptoms updated per 2024 consensus guidelines; references reviewed and updated; HCPCS coding updated — J1809 added and miscellaneous codes J3490 and C9399 removed.