WHA
 

Nitisinone Products

Indications for Prior Authorization

Generic nitisinone capsules
  • For diagnosis of Hereditary Tyrosinemia Type 1 (HT-1)
    Indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Criteria

Generic nitisinone

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Hereditary Tyrosinemia type 1 (HT-1)

  • Diagnosis of hereditary tyrosinemia type 1 (HT-1)
    • AND
  • Diagnosis confirmed by the presence of succinylacetone in the plasma or urine [1-3]
    • AND
  • Used in combination with dietary restriction of tyrosine and phenylalanine
    • AND
  • Prescribed by or in consultation with one of the following:
    • Gastroenterologist
    • Hepatologist
    • Other specialist with experience in treating inborn errors of metabolism
    AND
  • Applies to Nityr only; trial and intolerance to brand Orfadin
Generic nitisinone

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)
For diagnosis of Hereditary Tyrosinemia type 1 (HT-1)

  • Patient demonstrates a positive clinical response to therapy
    • AND
  • Applies to Nityr only; trial and intolerance to brand Orfadin

  • Guideline ID
    GL-498196

  • Formulary
    ehb

  • Effective date
    Feb 16, 2026

  • P&T approval date

P & T Revisions

2026-01-16, 2025-12-17, 2025-09-23, 2025-08-25, 2024-10-31, 2024-07-30, 2023-10-03, 2023-08-04, 2022-08-03, 2021-07-24, 2021-05-21, 2021-03-01, 2020-06-02, 2019-10-28

  1. Nityr prescribing information. Cycle Pharmaceuticals Ltd. Cambridge, UK. May 2024.
  2. Orfadin prescribing Information. Sobi Inc. Waltham, MA. November 2021.
  3. de Laet C, Dionisi-Vici C, Leonard JV, et al. Recommendations for the management of tyrosinaemia type 1. Orphanet J Rare Dis. 2013;8:8.
  4. Harliku prescribing information. Cycle Pharmaceuticals Ltd. Cambridge, UK. June 2025.
  5. ClinicalTrails.gov. Long-Term Study of Nitisinone to Treat Alkaptonuria. Available at: https://clinicaltrials.gov/study/NCT00107783?cond=NCT00107783&rank=1#participation-criteria. Accessed July 26, 2025.
  6. Alkaptonuria Society Alkaptonuria is a rare inherited disorder. Available at: https://akusociety.org/. Accessed July 26, 2025.
  7. Disorders of tyrosine metabolism: Clinical features, diagnosis, and management. Available at: https://www.uptodate.com/contents/disorders-of-tyrosine-metabolism-clinical-features-diagnosis-and-management?sectionName=ALKAPTONURIA&search=ALKAPTONURIA&topicRef=7196&anchor=H19&source=see_link#H19. Accessed July 26, 2025.
  8. Genetic and Rare Diseases Information Center. Alkaptonuria. Available at: https://rarediseases.info.nih.gov/diseases/5775/alkaptonuria. Accessed July 26, 2025.
  9. Wikipedia-Alkaptonuria. Available at: https://en.wikipedia.org/wiki/Alkaptonuria. Accessed July 26, 2025.
  10. Mishra, N. How to Diagnose Alkaptonuria: Urine Analysis and Genetic Testing. Available at: https://tap.health/how-to-diagnose-alkaptonuria-urine-analysis-and-genetic-testing/. Accessed July 26, 2025.
  11. Internal Research Response (RR) dated Sept 17, 2025 based on Clinical Consult from August 25, 2025. MD has experience in pediatrics, genetics, birth defects and metabolic disorder.
  1. In clinical trials, the diagnosis of alkaptonuria was based upon urinary HGA excretion greater than 0.4 g/24h. [5]
  2. Harliku is the only nitisinone product indicated for alkaptonuria (AKU). It was approved under a supplemental new drug application as a new indication for Nityr; however, the manufacturer has given it a unique brand name for this indication. Although there are no specific treatment guidelines for nitisinone products in the treatment of AKU, consultant feedback indicated that Harliku, Orfadin, and Nityr would be interchangeable, as they have the same active ingredient. [11]
  • 2026-01-16: New standalone EHB guideline containing covered products only.
  • 2025-12-17: Added IL statute operational note.
  • 2025-09-23: Annual review: No criteria changes.
  • 2025-08-25: Add drug Harliku to existing guideline
  • 2024-10-31: Added criterion to initial authorization and reauthorization: For Nityr only; trial and intolerance to brand Orfadin.
  • 2024-07-30: Annual review: No criteria changes. Updated references.
  • 2023-10-03: Program update to standard reauthorization language. No changes to clinical intent
  • 2023-08-04: Annual review: Added new 20mg nitisinone capsule (generic Orfadin) to mirror brand Orfadin criteria.
  • 2022-08-03: Annual review: No criteria changes. Updated references and background.
  • 2021-07-24: Updated references section.
  • 2021-05-21: Addition of EHB formulary to guideline, no changes to criteria
  • 2021-03-01: updated guideline to remove embedded step through Nityr for Brand Orfadin
  • 2020-06-02: Annual review: updated background & references
  • 2019-10-28: updated guideline to add generic nitisinone tablets