CurrentCareSourcePolicy N/A

Orfadin (nitisinone) — Pharmacy Coverage Criteria

Pharmacy benefit coverage and prior authorization requirements for Orfadin (nitisinone) for members in Arkansas PASSE; defines allowed site of service, quantity limits, diagnostic criteria (HT-1), and reauthorization conditions.

Policy Summary

PayerCareSource

PolicyOrfadin (nitisinone) — Pharmacy Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and document diagnosis, baseline succinylacetone, and a pre‑treatment eye exam in chart notes.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsinitial approval duration

2mg/kg/dayquantity limit

Homesite of service

Prior authorizationcoverage requirement

Coverage Criteria for Orfadin (nitisinone)

inv-01: Initial Therapy — Covered when ALL of the following are met for HT-1

Covered when ALL of the following are met for HT-1

HT-1 initial: Member has a diagnosis of hereditary tyrosinemia type 1 (HT-1);

HT-1 initial: Member has an eye exam (e.g., slit-lamp) performed and documented in chart notes prior to initiating treatment;

HT-1 initial: Member has a baseline succinylacetone level documented in chart notes;

HT-1 initial: Member will use the medication in combination with dietary restriction of tyrosine and phenylalanine;

Policy Summary

PayerCareSource

PolicyOrfadin (nitisinone) — Pharmacy Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and document diagnosis, baseline succinylacetone, and a pre‑treatment eye exam in chart notes.

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

HT-1 initial: Dosage does not exceed up to 1 mg/kg by mouth twice daily (up to 2 mg/kg/day);

If all requirements met, approve for 12 months.

inv-02: Reauthorization — Covered for reauthorization when ALL of the following are met

Covered for reauthorization when ALL of the following are met

HT-1 reauth: Chart notes demonstrate a positive response to therapy (e.g., reduction in succinylacetone level compared to baseline);

HT-1 reauth: Member continues dietary restriction of tyrosine and phenylalanine;

If reauthorization requirements met, approve for an additional 12 months.

Use of Orfadin (nitisinone) for indications other than those explicitly addressed in this policy is considered not medically necessary. Coverage is limited to the conditions and clinical scenarios described in this document; requests for treatment of diseases not listed here will be denied as not medically necessary.

Orfadin (nitisinone) is not medically necessary for the treatment of diseases other than those explicitly listed in this policy (Hereditary tyrosinemia type 1, HT-1). Requests for use outside of the listed diagnosis will not be approved.

Initial Therapy (HT-1)

inv-13: Initial therapy (HT-1) — Initial authorization criteria for HT-1

Initial authorization criteria for HT-1

Initial nitisinone for HT-1: Diagnosis of hereditary tyrosinemia type 1 (HT-1);

Initial nitisinone for HT-1: Documented eye exam (e.g., slit-lamp) performed and recorded in chart notes prior to initiating treatment;

Initial nitisinone for HT-1: Baseline succinylacetone level documented in chart notes;

Initial nitisinone for HT-1: Concurrent dietary restriction of tyrosine and phenylalanine;

Initial nitisinone for HT-1: Dosage limited to up to 1 mg/kg by mouth twice daily (up to 2 mg/kg/day);

Approve for 12 months if met.

Reauthorization / Continuation Requirements

inv-14: Reauthorization/Continuation — Reauthorization requirements for continued coverage

Reauthorization requirements for continued coverage

Continuation: Documentation of positive response to therapy (reduction in succinylacetone compared to baseline);

Continuation: Continued dietary restriction of tyrosine and phenylalanine;

Approve for additional 12 months if met.

Coding, Dosage, and Billing

Billing/NDCNDC

NDC required

Must use valid NDC code.

inv-06: Dosage

Dosage (oral)Up to 1 mg/kg by mouth twice daily (equivalent to up to 2 mg/kg/day).

Maximum daily doseEquivalent total daily dose = 2 mg/kg/day.

Administration frequencyTwice daily dosing (oral).

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Orfadin (nitisinone) under the pharmacy benefit. If the request is for brand-name Orfadin or the 10 mg capsule strength, follow the payer's DAW/Medical Necessity for DAW policy. Quantity limit: 2 mg/kg/day. Use a valid NDC when billing.

Benefit type: Pharmacy
Site of service allowed: Home
Quantity limit: 2 mg/kg/day
Use valid NDC
If brand Orfadin or 10 mg capsule requested, follow CareSource DAW policy

Note

Provider Actions

Ensure the following provider actions are completed and documented in the chart prior to initiation and for reauthorization as applicable.

Document eye exam (e.g., slit-lamp) prior to treatment initiation
Document baseline succinylacetone level in chart notes
Document dietary management with restriction of tyrosine and phenylalanine
For brand or 10 mg capsule strength, follow DAW/Medical Necessity for DAW policy

Documentation Required

Required Documentation

Clinical documentation must be submitted with the prior authorization request. Chart notes should include baseline and follow-up laboratory and exam findings, and ongoing dietary management.

Baseline succinylacetone level documented in chart notes
Documentation of an eye exam (e.g., slit-lamp) prior to treatment initiation
Chart notes demonstrating positive response on reauthorization (e.g., reduction in succinylacetone level compared to baseline)
Documentation that the member is following dietary restriction of tyrosine and phenylalanine

Denial Risk

Denial Triggers

Requests missing prior authorization or lacking required documentation will be denied. Denials may occur if diagnosis is not listed, baseline succinylacetone or eye exam is not documented, or dietary management is not documented.

Requests without prior authorization
Requests without documentation of required diagnosis
Requests without baseline succinylacetone level documented
Requests without documented eye exam prior to initiation
Requests without documentation of dietary restriction of tyrosine and phenylalanine

Step Therapy / DAW Rules

Step	Requirement / Action
If request is for brand Orfadin or 1 mg capsule strength
Follow the payer's 'Medical Necessity for DAW' policy on the CareSource website

Quantity Limits

inv-16: nitisinone (Orfadin)

Quantity limit2 mg/kg/day.

Benefit typePharmacy benefit (requires prior authorization).

Allowed site of serviceHome (dispensed under pharmacy benefit).

Allowed Site of Care

Note

Home use allowed

Orfadin (nitisinone) is allowed to be dispensed for use at home under the pharmacy benefit.

Definitions

inv-12: Baseline succinylacetone

DefinitionBaseline succinylacetone level: laboratory measurement documented before initiating nitisinone therapy to establish treatment response.

PurposeServes as the comparator for assessing treatment response on reauthorization (reduction in succinylacetone).

Documentation requirementMust be recorded in chart notes prior to starting nitisinone as part of initial authorization criteria.

Background

Hereditary tyrosinemia type 1 (HT-1) is an inherited metabolic disorder managed with nitisinone (Orfadin) in combination with dietary restriction of tyrosine and phenylalanine. Baseline and follow-up monitoring includes measurement of succinylacetone to assess treatment response, and an ophthalmologic evaluation (e.g., slit-lamp exam) should be documented prior to initiating therapy because of potential ocular effects. When the initial and follow-up criteria are met, therapy is approved for 12 months with reauthorization contingent on documented clinical benefit and continued dietary management.