CurrentColorado Rocky Mountain Health PlansPolicy N/A

Nitisinone (Orfadin®, Nityr®) — Prior Authorization and Coverage Criteria

Defines prior authorization requirements, documentation, and coverage criteria for nitisinone (Orfadin®, Nityr®) for members of Colorado Rocky Mountain Health Plans; applies to prescribers and pharmacies submitting PA requests. Maximum approval durations and dosing limits are specified.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyNitisinone (Orfadin®, Nityr®) — Prior Authorization and Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit a completed prior authorization form with supporting records and current weight; approvals issued up to 12 months (6 months if on or likely to need a liver transplant).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 moMax approval

6 moIf transplant-listed

2 mg/kgDose limit

60 caps / 90 mLPackaging

Coverage Criteria

Covered when ALL of the following are met

Coverage Criteria: 1) Patient has a diagnosis of hereditary tyrosinemia type I; 2) Dietary restrictions of tyrosine and phenylalanine alone are insufficient to maintain urinary succinylacetone at or below detectable levels (if dietary measures are sufficient, do not approve); 3) Provider submits required documentation, including completed PA form with prescriber signature and date, current weight in kg, and copies of medical records and most recent related labs; 4) Prescribed dose does not exceed 2 mg/kg for Orfadin or Nityr; 5) Approval duration: up to 12 months for standard patients, but limited to 6 months if the patient is on a liver transplant waiting list or is likely to become a transplant candidate within one year.dose and time-based

If the answer to question 2 on the PA form is 'Yes' (dietary restrictions sufficient), do not approve.

If the patient can be maintained on dietary restrictions alone, Orfadin or Nityr is not approved. This policy requires that dietary management (restriction of tyrosine and phenylalanine) must be insufficient to control disease biomarkers before nitisinone is authorized.

Use of Orfadin or Nityr is not medically necessary when dietary management alone maintains urinary succinylacetone at or below detectable levels. If dietary measures achieve control (question two = YES on the review criteria), do not approve nitisinone.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyNitisinone (Orfadin®, Nityr®) — Prior Authorization and Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit a completed prior authorization form with supporting records and current weight; approvals issued up to 12 months (6 months if on or likely to need a liver transplant).

SourceLink

On This Page

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Provider Actions & Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Use the completed Nitisinone (Orfadin®/Nityr®) PA form and fax to the Pharmacy PA fax number below. Approvals are issued for up to 12 months unless a shorter period applies (see items).

Fax completed PA form to: 1-866-940-7328
Pharmacy PA Call Center: 1-800-310-6826

Denial Risk

Dietary Management Assessment

The provider must confirm whether dietary restrictions of tyrosine and phenylalanine alone are sufficient to maintain urinary succinylacetone at or below detectable levels. If dietary management alone maintains acceptable levels (PA form question 2 = Yes), Orfadin or Nityr are not approved.

Documentation Required

Required Documentation

Submit a fully completed PA form including: recipient Medicaid ID, date of birth, recipient full name, prescriber name, prescriber NPI, prescriber phone and fax, pharmacy name, pharmacy Medicaid provider number, pharmacy phone and fax, answers to PA questions (diagnosis, dietary sufficiency, transplant listing, transplant candidacy), current weight in kg, prescriber signature and date. Include copies of all relevant medical records (diagnostic evaluations, recent chart notes) and the most recent related labs. Providers must retain documentation for five years.

Required for review: copies of medical records and most recent labs
Providers must retain copies of all documentation for five years

Background

Nitisinone (brand names Orfadin® and Nityr®) is indicated for management of hereditary tyrosinemia type I. Standard management includes strict dietary restriction of tyrosine and phenylalanine; nitisinone is used when dietary measures alone do not control relevant disease biomarkers (for example, urinary succinylacetone). Dosing is limited to 2 mg/kg. Approval durations are influenced by liver transplant status: approvals are generally for up to 12 months, but limited to 6 months if the patient is on a liver transplant list or is expected to become a transplant candidate within one year. Packaging notes: Orfadin is supplied as 60 capsules (cannot be repackaged) or a 90 mL suspension (4 mg/mL); Nityr is available as tablets.

Definitions

hereditary tyrosinemia type I — definition

DefinitionHereditary tyrosinemia type I — required diagnosis for coverage consideration.

Clinical contextNitisinone (Orfadin®, Nityr®) is used to treat hereditary tyrosinemia type I; dietary restriction of tyrosine and phenylalanine is part of management.

Coverage implicationIf patient can be maintained on dietary restrictions alone, Orfadin or Nityr is not approved.

Initial Therapy

Initial authorization requirements

Initial authorization: 1) Confirm diagnosis of hereditary tyrosinemia type I; 2) Document that dietary restrictions of tyrosine and phenylalanine alone are insufficient to maintain urinary succinylacetone at or below detectable levels (PA question 2 must be answered 'No'); 3) Submit completed PA form with prescriber's signature and date and current patient weight (kg); include copies of diagnostic evaluations, recent chart notes and most recent related labs; 4) Prescribed dose must not exceed 2 mg/kg; 5) Initial approval duration: up to 12 months, except when patient is on a liver transplant list or likely to become a transplant candidate within one year, in which case initial approval is limited to 6 months.dose and documentation

Incomplete forms may be returned; provider must retain documentation for five years.

Continuation Therapy

Continuation/renewal approvals

Renewal/Continuation: 1) Ongoing diagnosis of hereditary tyrosinemia type I; 2) Continued need demonstrated by clinical records and labs (dietary management remains insufficient); 3) Dose remains within the 2 mg/kg limit; 4) Renewal approval durations: up to 12 months for standard patients; if the patient is on a liver transplant waiting list or expected to require transplant within one year, renewal limited to 6 months.time-based

Follow same documentation retention and submission requirements as initial authorization.

Step Therapy / Treatment Sequence

Step	Requirement
1	Confirm that dietary restrictions of tyrosine and phenylalanine alone are insufficient to maintain urinary succinylacetone at or below detectable levels before approving nitisinone (Orfadin® or Nityr®). If the patient can be maintained on dietary restrictions alone, do not approve.

Quantity & Dosing Limits

Orfadin, Nityr — dosing and packaging

Dose limitLimit the dose to 2 mg/kg for Orfadin and Nityr.

Quantity limit unitExpressed as mg/kg per day (dose cap applies to daily dosing).

Approval contextDose limit is part of initial and continuation authorization criteria and must be met for approval.

Orfadin packaging noteOrfadin supplied as 60 capsules (HD polyethylene container; cannot be repackaged) or 90 mL suspension (4 mg/mL).

Site of Care

Note

Site‑of‑care — home allowed

No site‑of‑care restrictions are specified for nitisinone; home administration is acceptable per the policy.