Phenylketonuria treatment agent: Product Information

Product Information

Phenylketonuria treatment agent

PALYNZIQ® subcutaneous injection
2.5mg/10mg/20mg

PALYNZIQ® replaces phenylalanine hydroxylase (PAH), and unlike PAH, it metabolizes phenylalanine (Phe) into ammonia and cinnamic acid in a _BH4 -independent manner, thereby lowering blood Phe levels.

Generic Name

Pegvariase (genetically modified) preparations

Efficacy or effect

Phenylketonuria

Precautions Related to Indications or Effects

Application of this drug should be considered only when control of blood phenylalanine concentration is insufficient even with existing treatments.

Dosage and administration

Usually, adults are given a maintenance dose of 20 mg once a day as pegvariase (genetically modified) and administered subcutaneously.
However, the dose is increased in stages according to the following gradual escalation method, with a starting dose of 2.5 mg once a week.
If the effect of 20 mg once a day for a certain period of time is insufficient, the dose can be gradually increased to 40 mg or 60 mg, but the maximum dose is It is 60 mg.
The dosage should be adjusted as appropriate according to the patient's condition.

Gradual method up to 20 mg 1 time per day
Dosage/FrequencyDose and frequency of administration	Duration of administration
2.5 mg once weekly	4 weeks or more
2.5 mg twice a week	1 week or more
10 mg 1 time per week	1 week or more
10 mg twice a week	1 week or more
10 mg 4 times a week	1 week or more
10 mg 1 time per day	1 week or more
20 mg 1 time per day	―

Precautions related to usage and dosage

Until the maintenance dose is reached, the intake of phenylalanine from the diet should be managed to maintain a constant, the blood phenylalanine concentration should be measured at least once a month, and the patient's condition such as the development of hypersensitivity reactions should be carefully increased. After that, the patient's condition should be monitored and the blood phenylalanine concentration should be measured regularly to properly manage the blood phenylalanine concentration.
Increasing the dose to 40 mg may be considered when the effect is insufficient even after administering 20 mg once a day for at least 24 weeks. Depending on the patient's condition, if the effect of administering 20 mg once a day for more than 12 weeks is insufficient, the dose should be increased to 40 mg. It is possible to consider, but the necessity should be carefully judged based on the individual patient's condition.
An increase to 60 mg can be considered if the effect of administration of 40 mg 1 time per day for more than 16 weeks is insufficient.
If administration for a certain period of time does not provide sufficient effect, the necessity of continued administration should be determined by considering the benefits and risks.
Hypersensitivity reactions including anaphylaxis may develop with administration of this drug. In order to reduce symptoms, antihistamines and, if necessary, antipyretic analgesics should be administered in advance 2~3 hours before the start of administration of this drug. Pre-administration should be performed at least until the maintenance dose is reached, and administration at the maintenance dose should also be performed according to the patient's condition.
When starting administration, administer the drug under the supervision of a physician who can respond adequately in an emergency. Observe the patient thoroughly for at least 1 hour after administration.

Materials on Our Products

For more information on diseases and products, please refer to the following PDF documents.

Medical Reference

BioMarin Pharmaceutical Japan K.K. has established a medical information site " Medical Plus " For healthcare professionals .

Patient Support Program

'Palynziq Town' is a website for Phenylketonuria patients and their families receiving PALYNZIQ® .

Product Information : Phenylketonuria treatment agent

Product Information

Materials on Our Products

Medical Reference

Patient Support Program