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Prior authorization and medical necessity criteria for nitisinone products (Harliku, Orfadin, Nityr, generic nitisinone) for treatment of alkaptonuria (AKU) and hereditary tyrosinemia type 1 (HT-1), including initial and reauthorization criteria, prescriber requirements, and program-level notes. Authorization length is specified and some product-specific exclusions/plan variations noted.
Program created as new program in 2/2026.
Added educational note to AKU reauthorization section in 4/2026.
P&T approval dates recorded (2/2026, 4/2026) and effective date set to 5/1/2026.
Coverage stance: Covered with criteria for nitisinone products (Harliku, Orfadin, Nityr, and generic nitisinone) for the treatment of alkaptonuria (AKU) and hereditary tyrosinemia type 1 (HT-1). Prior authorization is required and authorizations are issued for 12 months. For AKU, initial authorization requires the patient to be ≥ 18 years old and meet specified biochemical or genetic thresholds; other prescriber and product-combination restrictions apply.
Initial Authorization for Harliku or Orfadin (AKU)
Covered when ALL of the following are met:
ALL of the following
ONE of
no concomitant nitisinone products
Initial Authorization for Nityr or Orfadin (HT-1)
Covered when ALL of the following are met:
ALL of the following
ONE of
no concomitant nitisinone products
Reauthorization for Harliku or Orfadin (AKU)
Covered when ALL of the following are met:
ALL of the following
no concomitant nitisinone products
Reauthorization for Nityr or Orfadin (HT-1)
Covered when ALL of the following are met:
ALL of the following
no concomitant nitisinone products
Prior authorization required
Prior authorization is required. Authorization will be issued for 12 months for both initial and reauthorization requests when all criteria are met.
Submit medical records/lab values or genetic testing
Providers must submit medical records such as chart notes and laboratory values or genetic testing confirming required biochemical thresholds or biallelic mutations (HGD for AKU or FAH for HT-1) as specified in the criteria.
Evidence of positive clinical response for reauthorization
For reauthorization, documentation of positive clinical response is required: for AKU, evidence such as reduced urinary HGA levels and/or improvement in joint symptoms; for HT-1, decreased urinary/plasma succinylacetone and alpha-1-microglobulin levels while on therapy.
Specialist prescriber requirement
Prescriptions must be written by or in consultation with a metabolic disease specialist; for AKU, a rheumatologist consultation is also specified in the criteria.
Product combination prohibition
Claims will only be approved if the requested nitisinone product is not used in combination with another nitisinone product.
Automated approvals allowed
UnitedHealthcare may approve initial and reauthorization requests based solely 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/or therapeutic class.
Plan-specific exclusions for Nityr
Nityr may be excluded from coverage for some plans; check member-specific plan benefits for exclusions or any tried/failed criteria that may apply.
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Evidence summary: Key trials cited include the SONIA 1 and SONIA 2 studies and the Orfadin pivotal studies referenced for AKU efficacy. The program notes limited Harliku-specific trials and states that Harliku approval was based on the Orfadin pivotal studies.
Background: Nitisinone is a hydroxyphenyl-pyruvate dioxygenase inhibitor. Nitisinone (generic Orfadin), Nityr, and Orfadin are indicated for treatment of HT-1 in adult and pediatric patients in combination with dietary restriction of tyrosine and phenylalanine. Harliku is indicated for the reduction of urinary homogentisic acid (HGA) in adult patients with alkaptonuria (AKU). The pivotal studies for Orfadin were used as the basis for FDA approval of Harliku; there were no additional clinical trials evaluating Harliku specifically.
Definitions: HGA = Homogentisic acid; HT-1 = Hereditary tyrosinemia type 1; AKU = Alkaptonuria.
Program created as new program (Program Number 2026 P 2412-2) noted in change control; Program type: Prior Authorization/Medical Necessity for nitisinone products.
Added educational note to AKU reauthorization section (non-material change) documenting background and educational statement regarding Harliku and AKU reauthorization criteria.
P&T approval dates recorded (2/2026 and 4/2026) for the program; administrative update aligning approvals.
Effective date for the program set to 2026-05-01 (administrative).