Pharmacy Prior Authorization Criteria

Nityr and Orfadin

Indications for Prior Authorization

Nityr (nitisinone) tablets

• 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.

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
AND
• 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

P & T Revisions

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

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.

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Revision History

• 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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