Nitisinone Products
Indications for Prior Authorization
Nityr (nitisinone) tablets
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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.
Brand Orfadin capsules, Brand Orfadin oral suspension, 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.
Harliku (nitisinone)
-
For diagnosis of Alkaptonuria (AKU)
Indicated for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU).
Criteria
Nityr*, Brand Orfadin, Generic nitisinone
*For patients who have difficulties swallowing intact tablets, including pediatric patients, the tablets can be disintegrated in water and administered using an oral syringe. If patients can swallow semi-solid foods, the tablets can also be crushed and mixed with applesauce. For preparation and administration instructions, see the full prescribing information [1].
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
- Applies to Nityr only; trial and intolerance to brand Orfadin
Nityr*, Brand Orfadin, Generic nitisinone
*For patients who have difficulties swallowing intact tablets, including pediatric patients, the tablets can be disintegrated in water and administered using an oral syringe. If patients can swallow semi-solid foods, the tablets can also be crushed and mixed with applesauce. For preparation and administration instructions, see the full prescribing information.
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
Harliku
Prior Authorization (Initial Authorization)
Length of Approval: 12 Month(s)
For diagnosis of Alkaptonuria (AKU)
- Diagnosis of alkaptonuria (AKU) AND
- Used for the reduction of urine homogentisic acid (HGA) AND
- Disease is confirmed by one of the following: [A]
- Presence of a mutation in the homogentisate 1,2-dioxygenase (HGD) gene as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
- Presence of elevated homogentisic acid (HGA) levels in urine greater than 0.4 g/24h
- Prescribed by or in consultation with one of the following:
- Rheumatologist
- Geneticist
- Specialist experienced in the treatment of inborn errors of metabolism
Harliku
Prior Authorization (Reauthorization)
Length of Approval: 12 Month(s)
For diagnosis of Alkaptonuria (AKU)
- Patient demonstrates positive clinical response to therapy (e.g., reduced levels of urinary HGA)
P & T Revisions
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
References
- Nityr prescribing information. Cycle Pharmaceuticals Ltd. Cambridge, UK. May 2024.
- Orfadin prescribing Information. Sobi Inc. Waltham, MA. November 2021.
- 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.
- Harliku prescribing information. Cycle Pharmaceuticals Ltd. Cambridge, UK. June 2025.
- 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.
- Alkaptonuria Society Alkaptonuria is a rare inherited disorder. Available at: https://akusociety.org/. Accessed July 26, 2025.
- 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.
- Genetic and Rare Diseases Information Center. Alkaptonuria. Available at: https://rarediseases.info.nih.gov/diseases/5775/alkaptonuria. Accessed July 26, 2025.
- Wikipedia-Alkaptonuria. Available at: https://en.wikipedia.org/wiki/Alkaptonuria. Accessed July 26, 2025.
- 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.
End Notes
- In clinical trials, the diagnosis of alkaptonuria was based upon urinary HGA excretion greater than 0.4 g/24h. [5]
Revision History
- 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
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