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# BioMarin’s sBLA for Palynziq for Expanded Use
- URL: https://www.fdaweb.com/biomarins-sbla-for-palynziq-for-expanded-use/
- Published: 2025-10-29T12:00:00.000Z
- Updated: 2026-09-14T15:25:17.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160129

FDA has accepted for priority review a BioMarin Pharmaceutical supplemental BLA for Palynziq (pegvaliase-pqpz) to expand the therapy’s use for treating adolescents aged 12–17 with phenylketonuria (PKU). The agency has set a user fee review action target date of 2/28/2026.

The submission is supported by data from the Phase 3 PEGASUS study, which demonstrated statistically significant reductions in blood phenylalanine (Phe) compared with diet alone, according to the company. The study also showed that Palynziq may allow adolescents to consume more dietary protein while achieving guideline-recommended or even normal Phe levels, it says.

Palynziq is currently approved as an enzyme substitution therapy for adults with PKU. The therapy substitutes the deficient PAH enzyme with a PEGylated form of phenylalanine ammonia lyase, helping metabolize Phe, BioMarin says.

PKU is described as a rare genetic disorder affecting the metabolism of phenylalanine, an amino acid found in most protein-containing foods. Without proper enzyme activity, Phe can accumulate to toxic levels, causing neurological complications such as intellectual disability, seizures, and behavioral issues, the company says.