Pharmacy Prior Authorization Criteria

Palynziq (pegvaliase-pqpz) - PA, NF

Indications for Prior Authorization

Palynziq (pegvaliase-pqpz)

• For diagnosis of Phenylketonuria (PKU)
  Indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.

Criteria

Palynziq

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of phenylketonuria (PKU)
AND
• Patient has uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management (e.g., phenylalanine restricted diet, Kuvan [sapropterin])
AND
• One of the following:
• Patient has had a trial and failure or intolerance to generic sapropterin
OR
• Patient is not a candidate for generic sapropterin therapy due to the presence of two null mutations in trans
AND
• Patient will have phenylalanine blood levels measured every 4 weeks until a maintenance dose is established and periodically thereafter [A]

Palynziq

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

• Patient has experienced an objective response to therapy, defined by one of the following [B, C]:
• At least a 20% reduction in blood phenylalanine concentrations from pre-treatment baseline
OR
• Blood phenylalanine concentrations less than or equal to 600 micromol/L
AND
• Patient will continue to have phenylalanine blood levels measured periodically during therapy [A]

Palynziq

Non Formulary

Length of Approval: 12 Month(s)

• Diagnosis of phenylketonuria (PKU)
AND
• Patient has uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management (e.g., phenylalanine restricted diet, Kuvan [sapropterin])
AND
• Submission of medical records (e.g., chart notes) or paid claims for one of the following:
• Patient has had a trial and failure or intolerance to generic sapropterin
OR
• Patient is not a candidate for generic sapropterin therapy due to the presence of two null mutations in trans
AND
• Patient will have phenylalanine blood levels measured every 4 weeks until a maintenance dose is established and periodically thereafter [A]

P & T Revisions

2024-11-04, 2023-10-03, 2023-04-06, 2022-10-31, 2021-10-05

References

1. Palynziq prescribing information. BioMarin Pharmaceutical Inc. Novato, CA. November 2020.
2. Vockley J, Andersson HC, Antshel KM, et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genet Med. 2014 Feb;16(2):188-200.

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End Notes

1. Patients should have blood phenylalanine (Phe) concentrations measured every 4 weeks after initiation of Palynziq (pegvaliase-pqpz), until a maintenance dosage is established. Periodic monitoring should continue after a maintenance dose is established [1].
2. Therapy should be discontinued in patients who do not achieve at least a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration less than or equal to 600 micromol/L after 16 weeks of continuous treatment with the maximum dosage of 40 mg once daily. Based on the recommended dosing regimen, patients could be evaluated for discontinuation after 49 weeks of therapy. This would allow for induction, titration, maintenance on 20 mg for 24 weeks, and maintenance on 40mg for 16 weeks.
3. The American College of Medical Genetics and Genomics guideline suggests blood Phe levels should be maintained in the range of 120–360 micromol/L for all patients [2].

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Revision History

• 2024-11-04: Annual review: No criteria changes.
• 2023-10-03: Annual review: Updated reauthorization approval length to 24 months.
• 2023-04-06: update guideline
• 2022-10-31: 2022 Annual Review - No changes to criteria
• 2021-10-05: 2021 Annual Review, no changes to criteria.

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