PALYNZIQ (pegvaliase-pqpz)
Phenylketonuria (PKU)
CommercialActive - recently approved for adolescents
Key Facts
Indication
Phenylketonuria (PKU)
Phase
Commercial
Status
Active - recently approved for adolescents
Company
About BioMarin Pharmaceutical
BioMarin Pharmaceutical is a publicly traded biotechnology company (NASDAQ: BMRN) focused on developing and commercializing innovative medicines for rare genetic diseases. With approximately 3,000 employees worldwide and operations in ~80 countries, the company has built a proven track record of bringing first-in-class therapies to market. Their distinctive approach to drug discovery leverages genetic insights to target underlying molecular mechanisms of disease, resulting in category-defining medicines that address significant unmet medical needs in rare disease communities.
View full company profileTherapeutic Areas
Other Phenylketonuria (PKU) Drugs
| Drug | Company | Phase |
|---|---|---|
| PTC923 | PTC Therapeutics | Phase 3 |