Important safety information

This page provides critical safety information for healthcare professionals involved in the Palynziq® (pegvaliase) treatment program for patients with phenylketonuria (PKU). It outlines procedures for reporting safety events and ensuring patient safety during treatment.

Definitions of Safety Events

  1. Adverse Event (AE): Any unwanted medical occurrence in a patient linked to the use of a medicinal product, regardless of whether it is considered related to the treatment with Pegvaliase.
  2. Adverse Drug Reaction (ADR): Any unwanted medical occurrence judged by the healthcare professional to have a reasonable causal relationship to Pegvaliase.
  3. Serious Adverse Event (SAE): An event meeting one or more of the following criteria:
    • Fatal
    • Life-threatening
    • Requires or prolongs hospitalization
    • Results in persistent or significant disability
    • Congenital malformation or birth defect
    • Important medical events that may require intervention to prevent serious outcomes​

Reporting Requirements

Healthcare professionals must report the following safety events within 1 business day of becoming aware:

  • All serious adverse events (SAEs)
  • Adverse drug reactions (ADRs) except for grade 1 and 2 injection site reactions
  • Anaphylaxis and acute systemic hypersensitivity reactions
  • Hypophenylalanemia (two consecutive measures ≤ 30µmol/L)
  • Infant safety events including congenital anomalies, serious adverse events, and specific reactions to Pegvaliase​

Laboratory Abnormalities

Clinically significant laboratory abnormalities must be reported if they are:

  • Accompanied by clinical symptoms
  • Resulting in a change in medication therapy
  • Suggestive of disease or organ toxicity​

Event Description and Follow-up

A detailed description of the event should include:

  • Clinical course and diagnostic results
  • Treatment and patient response
  • Relevant medical history and concomitant medications