Safety Event Definitions

The following information is for healthcare professionals only. If you are a potential PALomino study patient, please click here.

A Safety Reporting Guide is available to download by clicking here.

  • Adverse Event (AE) – An AE is any untoward medical occurrence in a patient temporally associated with the use of medicinal product, whether or not considered related to the treatment with pegvaliase.

    NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of pegvaliase

  • Adverse Drug Reaction (ADR) – Any untoward medical occurrence in a patient administered medicinal product that is judged by the reporting HCP (or Sponsor) as having a reasonable causal relationship to pegvaliase
    • Pre-existing medical conditions judged by the HCP to have worsened in severity or frequency or changed in character during the study and assessed as related to pegvaliase
  • Serious Adverse Event (SAE) – An SAE is any untoward medical occurrence that meets 1 or more of the following criteria:
    • Is fatal
    • Is life-threatening

      Note: life-threatening refers to an event that places the patient at immediate risk of death. This definition does not include a reaction that, had it occurred in a more severe form, might have caused death.

    • Requires or prolongs inpatient hospitalization
    • Results in persistent or significant disability or incapacity
    • Is a congenital malformation or birth defect in the child or fetus of a patient
    • Is an important medical event or reaction – that, based on medical judgment, may jeopardize the patient or require intervention to prevent one of the above consequences (e.g., anaphylaxis)

Clinically Significant Laboratory Abnormalities

  • Laboratory values are assessed by the patient’s HCP. Abnormal values (outside the reference range) are assessed as not related to the underlying disease or clearly attributable to another etiology. A clinically significant laboratory abnormality confirmed by repeat test per standard of care is defined as any one or more of the following:
    • accompanied by clinical symptoms
    • change in medication therapy
    • abnormality suggests disease or organ toxicity

Safety Event Evaluation

  • Seriousness Assessment – The reporting HCP will assess if an event should be classified as “serious” based on the seriousness criteria defined in the Safety Event Definitions above.
  • Severity Assessments – The reporting HCP will determine the severity of each reportable event. The CTCAE v5 will be used to assess event severity. To download the CTCAE v5, click here.
  • Causality Assessments – The reporting HCP will assess each event and report a causality of Related or Not Related to treatment with pegvaliase.
  • Clinical significance of reported Laboratory Results – The maternal and infant HCPs will use clinical experience to determine the significance of diagnostic test result abnormalities to determine if the event is reportable as defined in the Safety Event Definitions above.