Prescription medicine
Pegvaliase Brand names: Palynziq
Pegvaliase (Palynziq) is a prescription medicine. No generic is listed in FDA's NDC Directory.
- No generic listed
- Boxed warning
Check it at the source: the pegvaliase FDA label on DailyMed (effective March 2, 2026); every pegvaliase label on DailyMed.
Highlights of the record
FDA label of March 2, 2026; NDC Directory, Drugs@FDA and Orange Book, October 8, 2026
ANAPHYLAXIS Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment [see Warnings and Precautions (5.1) ]. Administer the initial dose of PALYNZIQ under the supervision of a healthcare provider equipped to manage anaphylaxis, and closely observe patients for at least 60 minutes following injection. Prior to self-injection, confirm patient competency with self-administration, and patient's and observer's (if applicable) ability to recognize signs and symptoms of anaphylaxis and administer epinephrine, if needed [see Dosage and Administration (2.5) ]. Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer epinephrine, and call for emergency medical support upon its use [see Warnings and Precautions (5.1) ]. Prescribe epinephrine to all patients treated with PALYNZIQ. Prior to the first dose, instruct the patient and observer (if applicable) how to recognize the signs and symptoms of anaphylaxis, how to properly administer epinephrine, and to seek immediate medical care upon its use. […]
Used for
From the label: indications and usagePALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.
Forms and strengths
- 2.5 mg/.5ml
- 10 mg/.5ml
- 20 mg/ml
Injection (solution).
On the market
- Generic approvals
- No generic approved
- Labelers listed (NDC)
- 1 of which 0 under generic applications, 1 under brand applications, repackagers included
Supply and safety
- Pharmacy cost (NADAC)
- Not in the survey
- Shortage (FDA)
- No current entry
- Recalls by its makers (FDA)
- No recall recorded
- FAERS reports
- 8,167 through July 30, 2026
What is pegvaliase used for?
From the label: indications and usagePALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. PALYNZIQ is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.
FDA pharmacologic class: Phenylalanine Metabolizing Enzyme.
Source: FDA drug label (Palynziq), BioMarin Pharmaceutical Inc., effective March 2, 2026 (SPL set 6dba844a-db02-44f8-8593-ce497ed9406c), via openFDA; the full label on DailyMed
What does the patient leaflet for pegvaliase say first?
The FDA-approved Medication Guide or Patient Information that comes with the medicine, in its own words.
From the patient leaflet: What is the most important information I should know about PALYNZIQ?PALYNZIQ can cause a severe allergic reaction (anaphylaxis). These reactions may be life-threatening and may happen any time during treatment with PALYNZIQ . Severe allergic reactions are a serious and common side effect of PALYNZIQ. You will get your first injection of PALYNZIQ under the supervision of a healthcare provider prepared to manage a severe allergic reaction. You will be closely watched for at least 1 hour after your injection for a severe allergic reaction. If you have a severe allergic reaction during treatment with PALYNZIQ, you will need to receive epinephrine and get emergency medical help right away. Your healthcare provider will decide if you (or your caregiver) are able to give PALYNZIQ injections, recognize the signs and symptoms of a severe allergic reaction, and use epinephrine and get emergency medical help, if needed. Your healthcare provider may ask an adult observer (or your caregiver) to be with you during and for at least 1 hour after each PALYNZIQ injection to watch for signs of a severe allergic reaction. If needed, they should be ready to give you epinephrine and get emergency medical help right away. […]
From the patient leaflet: How should I store PALYNZIQ?Store PALYNZIQ in the refrigerator at 36°F to 46°F (2°C to 8°C). If needed, you can store PALYNZIQ at room temperature between 68°F to 77°F (20°C to 25°C) for up to 30 days. Write the date that you remove PALYNZIQ from the refrigerator on the carton. If stored at room temperature, do not put PALYNZIQ back in the refrigerator. Keep PALYNZIQ in the original carton to protect from light. Do not freeze or shake PALYNZIQ. Throw away PALYNZIQ if it has been stored at room temperature for more than 30 days, or after the expiration date on the carton, whichever comes first. Keep PALYNZIQ and all medicines out of the reach of children.
Source: FDA drug label (Palynziq), BioMarin Pharmaceutical Inc., effective March 2, 2026 (SPL set 6dba844a-db02-44f8-8593-ce497ed9406c), via openFDA; the full label on DailyMed
Is there a generic for pegvaliase (Palynziq)?
No generic pegvaliase is approved (Drugs@FDA, October 8, 2026).
Source: Drugs@FDA, FDA NDC Directory and the Orange Book (openFDA bulk files, October 8, 2026)
What does the pegvaliase label say about other medicines?
The pegvaliase label names 2 other medicines in its interaction, contraindication or warning sections. The sentences are the labels' own; a label lists what its studies and reports found, not every interaction.
| Medicine named | Section | What the label says |
|---|---|---|
| Epinephrine | boxed warning | If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer epinephrine, and call for emergency medical support upon its use [see Warnings and Precautions (5.1) ]. |
| Medroxyprogesterone | drug interactions | In a single dose study of PALYNZIQ in adult patients with PKU, two patients receiving concomitant injections of medroxyprogesterone acetate suspension (a formulation containing PEG 3350) experienced a hypersensitivity reaction. |
Source: FDA drug label (Palynziq), BioMarin Pharmaceutical Inc., effective March 2, 2026 (SPL set 6dba844a-db02-44f8-8593-ce497ed9406c), via openFDA; the full label on DailyMed
What do FAERS reports show for pegvaliase?
The FDA Adverse Event Reporting System holds 8,167 reports that list pegvaliase among the medicines taken, through July 30, 2026, each a report of something that happened, not proof the medicine caused it; 12 are coded as a death and 301 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is.
Reports received each year
The most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026
What next?
- Pegvaliase side effects
- the label's list beside FAERS reports
- Check an interaction
- what two or more labels say about each other