Efficacy of Sapropterin For Phenylketonuria in Children, fin
A Study was conducted to assess the long-term safety and efficacy of sapropterin in Japanese patients with tetrahydrobiopterin (BH4)-responsive phenylketonuria (PKU).

This post-marketing surveillance study enrolled all patients with confirmed BH4-responsive PKU who were administrated Sapropterin. Patients were observed at least every 3 months during follow-up, with key data collected on treatment exposure/duration, effectiveness according to physician judgement, serum phenylalanine levels, and adverse events.

--Of 87 enrolled patients, 85 patients were included in the safety and efficacy analysis sets.

--Treatment started at age less than 4 years in 43 patients and the most common starting daily dose was 5–10 mg/kg (n=41) with the overall duration of treatment between 0.2 and 17.2 years.

--Serum phenylalanine levels according to loading tests reduced from a baseline level of 9.66 mg/dL by more than 30% in 84 patients.

--Treatment was deemed effective in 79 of 85 patients. One patient experienced an adverse drug reaction 50 days after the start of administration, which resolved without complications with continued treatment.

Finally, Sapropterin appears to be well tolerated and extremely effective in patients treated in a real-world situation, including children under the age of four and pregnant women.

Source: https://onlinelibrary.wiley.com/doi/10.1111/ped.14939?af=R