CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Nulibry (fosdenopterin) (Intravenous) — Coverage Criteria

Policy governing coverage and authorization criteria for Nulibry (fosdenopterin) IV for treatment of molybdenum cofactor deficiency (MoCD) Type A for Neighborhood Health Plan of Rhode Island members (Medicaid, Commercial, Medicare).

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyNulibry (fosdenopterin) (Intravenous) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateJul 1, 2021

Next Review DateN/A

Key ActionObtain prior authorization; initial approvals are for six months (three months one-time if diagnosis not yet confirmed).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsstandard initial authorization period

95 mgmaximum units per dose/day

J3490, C9399listed HCPCS billing codes

73129-0001-xxNDC (9.5 mg vial)

E61.5; E72.19covered diagnosis ICD-10 codes

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Coverage Criteria for Nulibry (fosdenopterin)

Initial Therapy

Covered when ALL of the following are met:

Universal requirements: Will not be used in combination with other substrate replacement therapy (e.g., recombinant cyclic pyranopterin monophosphate) AND must be prescribed by, or in consultation with, a specialist in medical genetics or pediatric neurology.

from Universal Criteria

Diagnosis confirmation: Patient has a diagnosis of MoCD Type A confirmed by molecular genetic testing showing a MOCS1 mutation OR has biochemical features suggestive of MoCD Type A (eg elevated urinary sulfites, low serum uric acid, elevated urinary xanthine and hypoxanthine) and is being treated presumptively while awaiting genetic confirmation.

diagnostic paths

Baseline data required: Documentation of baseline elevated urinary s-sulfocysteine (SSC) normalized to creatinine AND clinical notes documenting signs and symptoms of disease (eg seizure frequency/duration, growth, developmental milestones).

baseline measures required

Duration and limits: Initial approval for six months (three months one-time if diagnosis not confirmed).

authorization length

Renewal/Continuation Therapy

Renewal covered when ALL of the following are met:

Continued eligibility: Patient continues to meet universal and other indication-specific relevant criteria (including concomitant therapy requirements) and there is absence of unacceptable toxicity (eg severe phototoxicity or clinically significant infection).

safety requirement

Evidence of response: Either reduction in urinary SSC normalized to creatinine AND stabilization or improvement in one or more signs/symptoms (eg seizure frequency/duration, growth, developmental milestones) OR patient initiated therapy inpatient presumptively and diagnosis subsequently confirmed by genetic testing (documentation required).

response or retrospective confirmation paths

Timing for confirmation after presumptive start: For continued approval the diagnosis must be confirmed within the first 3 months of treatment.

time-bound confirmation

Use of Nulibry (fosdenopterin) in combination with any other substrate replacement therapy is prohibited. Examples include, but are not limited to, recombinant cyclic pyranopterin monophosphate. This restriction is part of the policy’s universal criteria and must be satisfied for coverage.

Requests will be denied when the patient lacks diagnostic confirmation or required baseline biochemical and clinical documentation and is not being treated presumptively while awaiting confirmation. Covered patients must either have molecular genetic confirmation of MoCD Type A (MOCS1 mutation) or biochemical features strongly suggestive of MoCD Type A and be started presumptively. In all cases, providers must submit baseline laboratory evidence of an elevated urinary s-sulfocysteine (SSC) normalized to creatinine and clinical notes documenting relevant signs and symptoms (for example, seizure frequency/duration, growth, and developmental milestones).

Coding and Dose Limits

HCPCS CodesHCPCS

J3490	Unclassified drugs
C9399	Unclassified drugs or biologicals (hospital outpatient use)

NDCNDC

73129-0001-xx

Nulibry 9.5 mg single-dose vial as a lyophilized powder for injection

Covered Diagnosis Codes (ICD-10)ICD-10Covered

E61.5	Molybdenum deficiency
E72.19	Other disorders of sulfur-bearing amino-acid metabolism

inv-08: Maximum daily dose — single-value key/value highlighting 95 mg daily

Maximum daily dose95 mg daily

Unit typeHCPCS unit (per dose and over time)

Dosing frequencyOnce daily IV infusion (age- and weight-based dosing per Dosage/Administration)

Provider Requirements and Authorization

Prior Authorization

Prior Authorization and Approval Period

Prior authorization is required. Initial approvals are for six months. If diagnosis is not confirmed at time of request, initial approval will be for three months (one-time only). Medicare members who previously received the medication within the past 365 days are exempt from step therapy requirements.

Initial approval: 6 months
If diagnosis not confirmed: 3 months (one-time)
Medicare prior-user exemption: previous receipt within 365 days

Step Therapy

Step Therapy and Concomitant Therapy

Step therapy does not apply to Medicare members who have received this medication within the past 365 days. Therapy with this product must not be used in combination with other substrate replacement therapies (e.g., recombinant cyclic pyranopterin monophosphate).

Medicare exemption: prior receipt within 365 days

Background

Nulibry (fosdenopterin) received U.S. Food and Drug Administration approval for reduction of mortality risk in patients with molybdenum cofactor deficiency (MoCD) Type A. It is administered once daily by intravenous infusion with age- and weight-based dosing. The indication encompasses patients with genetically confirmed disease (MOCS1 mutation) as well as those started presumptively based on characteristic biochemical abnormalities while genetic testing is pending; baseline and follow-up biochemical and clinical data are required to support continued therapy.

Definitions

inv-14: MoCD Type A — definition and diagnostic criteria (MOCS1 mutation / biochemical abnormalities)

DefinitionMolybdenum cofactor deficiency Type A (MoCD Type A) — a genetic metabolic disorder diagnosed by a mutation in the MOCS1 gene or by characteristic biochemical abnormalities (elevated urinary sulfites, low serum uric acid, elevated urinary xanthine and hypoxanthine).

Diagnostic genetic criterionConfirmation by molecular genetic testing demonstrating a pathogenic MOCS1 mutation.

Biochemical diagnostic criterionBiochemical features suggestive of MoCD Type A (eg elevated urinary sulfites, low serum uric acid, elevated urinary xanthine and hypoxanthine) — may justify presumptive treatment while awaiting genetic confirmation.

Required baseline dataBaseline elevated urinary s-sulfocysteine (SSC) normalized to creatinine and clinical documentation of signs/symptoms (eg seizure frequency/duration, growth, developmental milestones).

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyNulibry (fosdenopterin) (Intravenous) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateJul 1, 2021

Next Review DateN/A

Key ActionObtain prior authorization; initial approvals are for six months (three months one-time if diagnosis not yet confirmed).

SourceLink

On This Page

Prohibited concomitant therapy: other substrate replacement therapies

Denial Risk

Diagnostic Confirmation Required

Requests must include diagnostic confirmation of MoCD Type A by molecular genetic testing showing a pathogenic or likely pathogenic mutation in MOCS1, or, if genetic results are pending, biochemical evidence strongly suggestive of MoCD Type A (e.g., elevated urinary sulfites, low serum uric acid, elevated urinary xanthine and hypoxanthine). If treated presumptively while awaiting genetic confirmation, documentation of the plan to obtain confirmatory testing is required.

Required diagnostic confirmation: molecular genetic testing showing mutation in MOCS1 OR biochemical features suggestive of MoCD Type A
Permissive presumptive treatment: allowed while awaiting genetic confirmation but genetic testing must be pursued

Documentation Required

Baseline and Follow-up Documentation

Providers must supply baseline and follow-up documentation with the request and for renewals. Baseline documentation required: urinary S-sulfocysteine (SSC) normalized to creatinine, and clinical notes describing signs and symptoms (including seizure frequency/duration, growth, developmental milestones). For renewal, provide evidence of response such as reduction in urinary SSC normalized to creatinine and stabilization or improvement in clinical signs/symptoms. If therapy was started presumptively inpatient, submit confirmatory genetic testing results when available; diagnosis must be confirmed within the first 3 months of treatment for continued approval.

Baseline labs: urinary SSC normalized to creatinine (documentation required)
Baseline clinical documentation: signs/symptoms, seizure frequency/duration, growth, developmental milestones
Renewal evidence: reduction in urinary SSC normalized to creatinine AND stabilization or improvement in clinical signs/symptoms
If inpatient presumptive start: submit genetic confirmation; diagnosis must be confirmed within first 3 months for continued approval