Can you name trials supporting sapropterin use?

What trials tested sapropterin for phenylketonuria?

Several randomized controlled trials evaluated sapropterin as a treatment for phenylketonuria. One study measured blood phenylalanine levels in patients who received 20 mg/kg/day of sapropterin for six weeks, showing a significant reduction compared to placebo [1].

What happened in the long-term extension study?

The extension study followed patients who responded to sapropterin for an average of 13 months. Those who kept taking 5–20 mg/kg/day showed sustained reductions in blood phenylalanine levels while maintaining or improving dietary protein tolerance [2].

How did sapropterin affect children under four years old?

A dedicated pediatric study enrolled patients aged zero to four years. These children received 20 mg/kg/day for six weeks and recorded a reduction in blood phenylalanine levels [3].

What does the meta-analysis say about overall effectiveness?

A meta-analysis of 12 trials concluded that sapropterin reduces blood phenylalanine levels and increases natural protein tolerance in tetrahydrobiopterin-responsive patients [4].

When does the patent for sapropterin dihydrochloride expire?

The U.S. patent covering the crystalline form of sapropterin dihydrochloride held by BioMarin expires in 2025 [5].