What patient demographics did sapropterin trials mainly cover?

Who participated in the main sapropterin trials?

The trials that supported approval of sapropterin included mostly children and adults who had phenylketonuria (PKU) and maintained blood phenylalanine levels above 600 µmol/L despite dietary treatment.

Patients ranged in age from 4 years upward, with several studies focusing on children under 18 while others included mixed adult and younger populations.

Women of childbearing potential were also tested, but pregnancy itself was not studied.

What age groups showed the strongest response?

Children between 4 and 12 years old responded more reliably to sapropterin than older patients.