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# BioMarin Files BLA for Phenylketonuria Therapy
- URL: https://www.fdaweb.com/biomarin-files-bla-for-phenylketonuria-therapy/
- Published: 2017-06-30T12:00:00.000Z
- Updated: 2026-09-14T22:32:43.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5139069

BioMarin Pharmaceutical Inc. has filed a BLA for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, indicated for reducing blood phenylalanine (Phe) levels in adult patients with Phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing therapy. “Pegvaliase has been shown to lower blood Phe levels, which was the primary endpoint for registration of the only therapy currently approved to treat PKU,” the company says. 

Biomarin says pegvaliase works by substituting the deficient PAH enzyme in PKU with the PEGylated version of the enzyme phenylalanine lyase, to break down Phe. “In clinical studies, treatment with subcutaneous pegvaliase substantially reduced blood Phe compared to placebo using a randomized withdrawal study design, and led to long-term maintenance of Phe reduction in the majority of adult patients with PKU,” it says. “Pegvaliase was administered using a dosing regimen that achieved a manageable safety profile, consisting primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures effective in clinical trials will be proposed.”