Clinical Development
By streamlining clinical and regulatory development, BioMarin is providing rapid access to treatment to patients around the world suffering from rare inherited genetic diseases. The company is focused on accelerating the regulatory approval process by maximizing integrated product development strategies and leveraging the expertise of collaborative, cross-functional teams of clinical and regulatory professionals. Currently, BioMarin has an exciting range of breakthrough drug candidates now in clinical development: 6R-BH4 for cardiovascular and sickle cell diseases and PEG-PAL (PEGylated recombinant phenylalanine ammonia lysase) for PKU, an enzyme substitution therapy which may have the potential to treat the entire spectrum of PKU patients. A variety of other promising preclinical programs are also being pursued with the goal of filing one IND per year to ensure continued growth.
Click on indications to learn more about each product candidate.
