SFDA Grants Orphan Drug Designation to “Sephience”
2026-01-28
The Saudi Food and Drug Authority (SFDA) has granted PTC Therapeutics orphan drug designation to Sephience (sepiapterin), which is indicated for the treatment of hyperphenylalaninaemia (HPA) in patients with phenylketonuria (PKU).
Registration is Subjected to Full Scientific Evaluation