trending Market Intelligence /marketintelligence/en/news-insights/trending/ejybp5hhv_g8uha1i8yauw2 content esgSubNav
In This List

BioMarin's genetic disease drug Palynziq gets US FDA approval

Blog

A Pharmaceutical Company Capitalizes on M&A Activity with Brokerage Research

Blog

2021 Year in Review: Highlighting Key Investment Banking Trends

Blog

Insight Weekly: US stock performance; banks' M&A risk; COVID-19 vaccine makers' earnings

Blog

Global M&A By the Numbers: Q3 2021


BioMarin's genetic disease drug Palynziq gets US FDA approval

The U.S. Food and Drug Administration approved BioMarin Pharmaceutical Inc.'s Palynziq, or pegvaliase, injection to reduce blood amino acid phenylalanine concentration in adult patients with the genetic disease phenylketonuria.

Phenylketonuria is marked by an inability to break down phenylalanine, an amino acid that is found in all forms of protein. The rare genetic disease manifests at birth and results in a variety of cumulative toxic effects on the brain.

Palynziq, an enzyme substitution therapy that helps the body break down phenylalanine, is the U.S. biotechnology company's second approved treatment for phenylketonuria.

The Palynziq approval is indicated for patients who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.

BioMarin plans to begin marketing of Palynziq immediately after its launch in the U.S., which is expected by the end of June.

San Rafael, Calif.-based BioMarin Pharmaceutical filed an FDA biologics license application for Palynziq to treat phenylketonuria in June 2017 and in August 2017 the application was granted a priority review.

The European Medicines Agency accepted BioMarin's marketing authorization application for Palynziq in March.