The FDA approved BioMarin Pharmaceutical's therapy for adults with the rare inherited disorder phenylketonuria, the drugmaker announced May 24.
Here are three things to know:
1. Patients with PKU are unable to break down the amino acid phenylalanine in their blood, which causes a dangerous accumulation of the substance. The disease affects about 1 in 12,500 live births in the U.S. annually.
2. BioMarin's Palynziq injection represents the first FDA-approved enzyme substitution therapy to help the body break down Phe.
"The goal in treating PKU is to keep blood Phe levels within the range set in the medical guidelines, as elevation of Phe can be toxic and damaging to the brain," said Cary Harding, MD, a professor at Portland-based Oregon Health & Science University and investigator in a phase 3 clinical trial for the drug. "Palynziq provides another much needed tool for us to help adult patients control their Phe levels, which previously had not been achievable for many adults living with the condition."
3. BioMarin expects Palynziq to hit the market by the end of June.
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