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# BioMarin Planning NDA for Genetic Disorder
- URL: https://www.fdaweb.com/biomarin-planning-nda-for-genetic-disorder/
- Published: 2016-03-22T12:00:00.000Z
- Updated: 2026-09-14T20:50:35.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5135156

> BioMarin says it will file by the end of the year an NDA for pegvaliase and its use in treating genetic disorder phenylketonuria (PKU). The decision to submit an NDA follows just-announced Phase 3 results from the PRISM-2 study, which showed the drug significantly lowered levels of phenylalanine, or Phe, in the blood of PKU patients by 62%. Pegvaliase substitutes the PAH enzyme in PKU by breaking down Phe and is being developed as a potential treatment for adults with inadequately controlled blood Phe levels, according to the company.