Summary:
- The U.S. Food and Drug Administration (FDA) has approved Biomarin's Palynziq (pegvaliase-pqpz) for the treatment of adolescents 12 years of age and older with phenylketonuria (PKU).
- PKU is a rare, inherited metabolic disorder that can lead to intellectual disability and other serious health problems if left untreated. Palynziq helps manage PKU by breaking down the amino acid phenylalanine, which builds up in people with PKU.
- This approval expands the use of Palynziq, which was previously approved for adults with PKU. The availability of this treatment option for adolescents is important, as PKU can have significant impacts on growth and development during the teenage years.