Phenylketonurias

Top drugs studied for this disease

EMA approval · 2026-01-07
EMA Authorised: Kuvan (sapropterin dihydrochloride) — BioMarin International Limited
The European Medicines Agency has authorised Kuvan (sapropterin dihydrochloride) for phenylketonurias. The marketing authorization holder is…
CLINICAL_TRIALS trial_result · 2025-11-28
Clinical Trial Update: Evaluation of PKU Explore France [NA]
A clinical trial evaluating PKU Explore France for phenylketonurias, sponsored by Vitaflo International, Ltd., has completed (NCT04898829).