Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data

ABSTRACT:

Background

Phenylketonuria (PKU) is an inborn metabolic error characterized by a deficiency of the enzyme required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods. Pegvaliase (Palynziq®) is an enzyme substitution therapy approved for adults with PKU who have inadequate blood phenylalanine control (≥600 μmol/L) on existing management.

Objective

To characterize the treatment, discontinuation, and dosing patterns in patients treated with pegvaliase in real-world practice settings in the United States following commercial availability in 2018.

Study design

Retrospective cohort study using BioMarin's proprietary drug dispense database associated with the pegvaliase REMS program.

Methods

Sample construction i

SUBMITTER: Lah M 

PROVIDER: S-EPMC9513695 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature