The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as phenylketonuria (PKU).

Dec 21, 2023 · The U.S. Food and Drug Administration (FDA) has approved the drug sapropterin dihydrochloride (Kuvan) for the treatment of PKU. Kuvan is a form of BH4, which is a substance in the body that helps break down phenylalanine.

Phenylketonuria (PKU) is a rare hereditary condition in which the amino acid phenylalanine is not properly metabolized. PKU can cause severe mental retardation if not treated.

Stop injecting PALYNZIQ and get emergency medical care right away if you have any of the following symptoms: Keep the auto-injectable epinephrine with you at all times during treatment with PALYNZIQ. Read the Patient Information that comes with the auto-injectable epinephrine that your healthcare provider prescribes for you for more information.

May 13, 2022 · PKU is caused by a change in the gene that helps create an enzyme needed to break down phenylalanine. Treatment includes a special diet and medication.

Phenylketonuria (PKU) is a genetic condition that passes to children from their parents in an autosomal recessive pattern. This means that babies receive one copy of the mutated gene that causes PKU from each parent during conception.

Aug 8, 2023 · Phenylketonuria (PKU) is an inborn error of metabolism (IEM) most often caused by missense mutations in the gene encoding phenylalanine hydroxylase (PAH), which catalyzes the hydroxylation of phenylalanine (Phe) to generate tyrosine (Tyr).

Jan 18, 2024 · The Food and Drug Administration (FDA) has approved two drug treatments for PKU. Sapropterin (Kuvan) is an oral drug that acts as the cofactor for the enzyme affected by PKU. It can stimulate ...

Oct 7, 2020 · Palynziq is indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 μmol/L on existing management.

Apr 2, 2025 · DRUG INTERACTIONS. Effect of PALYNZIQ on Other PEGylated Products. 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. One of the two patients experienced anaphylaxis