Medications for Phenylketonuria (PKU)
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Phenylketonuria (PKU).
Found 2 Approved Drugs for Phenylketonuria (PKU)
Sephience
Generic Name
Sepiapterin
Sephience
Generic Name
Sepiapterin
Form: Powder
Method of administration: Oral
FDA approval date: July 28, 2025
SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)-restricted diet. SEPHIENCE is a phenylalanine hydroxylase (PAH) activator indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)- restricted diet. ( 1 )
Palynziq
Generic Name
Pegvaliase-Pqpz
Palynziq
Generic Name
Pegvaliase-Pqpz
Form: Injection
Method of administration: Subcutaneous
FDA approval date: May 24, 2018
Classification: Phenylalanine Metabolizing Enzyme
Palynziq is 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. Palynziq is a phenylalanine (Phe)‑metabolizing enzyme indicated to reduce blood Phe concentrations in adult patients with phenylketonuria who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. ( 1 )
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