PTC923
Phenylketonuria (PKU)
Phase 3Clinical
Key Facts
About PTC Therapeutics
PTC Therapeutics is a patient-centric, publicly traded biopharma leader dedicated to translating groundbreaking science into life-changing treatments for rare diseases. Its key achievements include commercializing a series of 'firsts' in rare disease treatment and building a diversified pipeline targeting neurological and metabolic disorders. The company's strategic direction is fueled by its deep expertise in two core scientific platforms—splicing modifiers and inflammation/ferroptosis inhibitors—and a robust global commercial infrastructure to support its mission of delivering transformative medicines worldwide.
View full company profileTherapeutic Areas
Other Phenylketonuria (PKU) Drugs
| Drug | Company | Phase |
|---|---|---|
| PALYNZIQ (pegvaliase-pqpz) | BioMarin Pharmaceutical | Commercial |