Palynziq (pegvaliase-pqpz) is a prescription medication used to lower blood levels of phenylalanine (Phe) in adults with phenylketonuria (PKU) who have uncontrolled blood Phe levels above 600 micromol/L (10 mg/dL) on their current treatment. PKU is a rare genetic disorder that prevents the body from breaking down the amino acid phenylalanine. Phenylalanine can build up in the blood and cause serious health problems, such as intellectual disability, seizures, and behavioral problems.
Palynziq is an enzyme replacement therapy (ERT) drug. It works by replacing the missing or defective phenylalanine hydroxylase (PAH) enzyme, which is responsible for breaking down phenylalanine. Palynziq is given as a once-daily subcutaneous injection.
How effective is Palynziq?
In clinical studies, Palynziq was shown to be effective in lowering blood Phe levels in patients with PKU. In one study, Palynziq reduced blood Phe levels by a median of 60% after 12 weeks of treatment.
What are the side effects of Palynziq?
The most common side effects of Palynziq are:
- Injection site reactions
- Headache
- Fatigue
- Nausea
- Vomiting
- Diarrhea
- Constipation
- Abdominal pain
- Back pain
- Muscle pain
- Upper respiratory tract infection
Palynziq can also cause more serious side effects, such as:
- Allergic reactions, including anaphylaxis (a life-threatening allergic reaction)
- Decreased liver function
- Decreased kidney function
- Infusion-related reactions
- Increased risk of infection
Who should not take Palynziq?
Palynziq should not be taken by people who are allergic to pegvaliase-pqpz or any of the other ingredients in Palynziq.
Important safety information
It is important to tell your doctor about all of the medications you are taking before starting Palynziq. Palynziq can interact with other drugs, such as certain antibiotics and anticonvulsants.
Palynziq should be used with caution in people with liver disease or kidney disease.
Palynziq can cause serious side effects, such as allergic reactions and decreased liver function. It is important to monitor your liver function regularly while taking Palynziq.
If you are considering taking Palynziq, talk to your doctor about the risks and benefits of the medication.
James P. –
The support and education I received along with Palynziq made a big difference. The treatment itself has been effective at managing my PKU, reducing symptoms, and improving my quality of life. I feel empowered knowing I have this option available.
Monica S. –
Palynziq has helped me lower my phenylalanine levels consistently. I’m able to enjoy foods I previously avoided and feel more balanced overall. It’s not a quick fix, but with patience and support, it really works. I recommend it to anyone with PKU seeking better control.
Daniel B. –
After years of struggling with PKU, Palynziq has given me hope. My cognitive clarity has improved, and I feel less anxious about my diet. The side effects were manageable, and my healthcare team guided me through the process. So glad this treatment exists!
kmitchell_pkujourney –
Palynziq has been a breakthrough for me in managing phenylketonuria. Before, my diet was so restrictive, and I struggled to keep my phenylalanine levels down. Since starting Palynziq, my blood levels are much more stable, and I have more freedom in my diet. The injection process was intimidating at first, but now it’s routine.