CurrentUnitedHealthcarePolicy N/A

Harliku (nitisinone) prior authorization

Defines prior authorization and medical necessity criteria for Harliku (nitisinone) for treatment of alkaptonuria in adults; applies to UnitedHealthcare benefit plans and providers requesting authorization. Affects prescribers, pharmacists, and utilization management staff.

Policy Summary

PayerUnitedHealthcare

PolicyHarliku (nitisinone) prior authorization

Policy CodePolicy N/A

Change TypeNew policy

Effective DateFeb 1, 2026

Next Review DateN/A

Key ActionSubmit prior authorization with medical records confirming diagnosis and diagnostic confirmation (urinary HGA > 0.4 g/24 hr or biallelic HGD mutation).

SourceLink

POLICY UPDATE CHANGES

New program established for prior authorization/medical necessity of Harliku (nitisinone).

12 monthsauthorization period

≥18minimum age

>0.4 g/24 hrHGA threshold

Specialistsprescriber specialties

3prohibited combinations

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Coverage Criteria for Harliku (nitisinone)

Initial Therapy

Covered when ALL of the following are met

Initial Authorization for Harliku: a) Diagnosis of alkaptonuria; b) patient is >= 18 years old; c) submission of medical records confirming one of: (1) urinary homogentisic acid (HGA) excretion > 0.4 g/24 hours OR (2) biallelic mutation in HGD confirmed by genetic testing; d) Harliku will not be used in combination with generic nitisinone, Nityr, or Orfadin; e) prescribed by or in consultation with a geneticist, metabolic disease specialist, or rheumatologist.

Authorization will be issued for 12 months.

Notes: State mandates, federal requirements, and member benefit plan coverage may affect approval; other policies/utilization programs may apply.

Continuation/Reauthorization

Reauthorization covered when ALL of the following are met

Reauthorization for Harliku: a) Documentation of positive clinical response to Harliku therapy (e.g., reduced urinary HGA levels, improvement in joint symptoms); b) Harliku will not be used in combination with generic nitisinone, Nityr, or Orfadin; c) prescribed by or in consultation with a geneticist, metabolic disease specialist, or rheumatologist.

Authorization will be issued for 12 months.

Use of Harliku concurrently with other nitisinone products is prohibited. Specifically, combination therapy with generic nitisinone, Nityr, or Orfadin is not allowed and will trigger denial of authorization.

Requests for Harliku that lack required diagnostic confirmation or that are for patients under 18 years of age do not meet medical necessity criteria. Required diagnostic confirmation must include either urinary HGA excretion > 0.4 g/24 hours or documentation of a biallelic HGD mutation. The policy applies only to adults aged ≥ 18 years.

Initial Therapy

Initial therapy

Initial coverage criteria

Initial therapy: a) Diagnosis of alkaptonuria; b) patient is >= 18 years old; c) diagnostic confirmation by one of: urinary HGA excretion > 0.4 g/24 hours OR biallelic HGD mutation on genetic testing; d) not used in combination with other nitisinone products (generic nitisinone, Nityr, Orfadin); e) prescribed by or in consultation with a geneticist, metabolic disease specialist, or rheumatologist.

Authorization will be issued for 12 months.

Reauthorization / Continuation Criteria

Reauthorization

Reauthorization requires demonstration of benefit and continued adherence to prescribing restrictions.

Reauthorization: Documentation of positive clinical response to Harliku (e.g., reduced urinary HGA, improved joint symptoms); continued avoidance of combination with other nitisinone products (generic nitisinone, Nityr, Orfadin); ongoing prescription by or in consultation with a geneticist, metabolic disease specialist, or rheumatologist.

Authorization will be issued for 12 months.

Coding and Diagnostic Thresholds

No explicit CPT/HCPCS/NDC codes listedmixed

No codes listed

inv-06: Urinary homogentisic acid (HGA) excretion threshold

HGA excretion threshold> 0.4 g/24 hours

Measurement typeUrinary homogentisic acid (HGA) excretion over 24 hours

Use for diagnosis and eligibilityRequired as part of initial diagnostic confirmation for alkaptonuria to qualify for Harliku prior authorization

Provider Actions, Authorization & Documentation

Prior Authorization

Initial Prior Authorization Required

Prior authorization required for Harliku. Initial prior authorization will be approved when all specified criteria are met, including diagnosis of alkaptonuria, age ≥ 18 years, submission of diagnostic documentation confirming urinary HGA > 0.4 g/24 hr or biallelic HGD mutation, no concurrent use with listed alternatives, and prescribing by or in consultation with an appropriate specialist. Authorization is issued for 12 months.

Diagnosis of alkaptonuria
Patient is ≥ 18 years old
Submit medical records confirming: urinary HGA > 0.4 g/24 hr OR biallelic HGD mutation by genetic testing
Harliku not to be used with generic nitisinone, Nityr, or Orfadin
Prescribed by or in consultation with a geneticist, metabolic disease specialist, or rheumatologist
Authorization duration: 12 months

Prior Authorization

Reauthorization Requirements

Reauthorization requires documentation of continued benefit from Harliku therapy. Approvals granted when all reauthorization criteria are met, including documentation of clinical improvement and adherence to prescribing restrictions. Authorization is issued for 12 months.

Background

Harliku (nitisinone) is a hydroxyphenyl-pyruvate dioxygenase (HPPD) inhibitor indicated to reduce urinary homogentisic acid (HGA) in adults with alkaptonuria (AKU). Reduction in urinary HGA is used as a biochemical marker of response to therapy; prescribing should be coordinated with clinicians experienced in genetic or metabolic disorders.

Definitions

inv-14: Alkaptonuria — definition and pathophysiology

DefinitionAlkaptonuria (AKU) is a rare inherited disorder caused by deficiency of homogentisate 1,2-dioxygenase (HGD), leading to accumulation and urinary excretion of homogentisic acid (HGA).

PathophysiologyDeficiency of HGD enzyme causes impaired breakdown of homogentisic acid, resulting in elevated HGA that deposits in connective tissues and is excreted in urine.

Clinical markerElevated urinary HGA is the biochemical marker used to confirm diagnosis and monitor response to therapy (Harliku reduces urinary HGA).

inv-15: Diagnostic confirmation for AKU — urinary HGA > 0.4 g/24 hours or biallelic pathogenic HGD variants

Acceptable diagnostic confirmationsUrinary HGA excretion > 0.4 g/24 hours

Genetic confirmation alternative

Step Therapy / Automated Review

Step	Requirement	Notes
1
Automated approval or review may be used to approve initial or reauthorization requests based on prior claim/medication history, diagnosis codes (ICD-10), and/or claim logic.
Use of automated approval and re-approval processes varies by program and therapeutic class; no specific step agent or prerequisite medication is listed.

Site of Care / Supply Notes

Billing Rule

Supply limits may apply; site-of-care restrictions not specified

Supply limits may apply for Harliku; no specific site-of-care restrictions are specified in the policy (site listed as infusion center with possible supply limits).

Policy notes that supply limits may be in place
No explicit site-of-care restrictions provided