Biomarin’s hemophilia drug nets PRIME status in Europe

BioMarin Pharmaceutical’s treatment for hemophilia has been accepted for PRIME (Priority Medicines) scheme from the European Medicines Agency (EMA).

BioMarin’s investigational gene therapy treatment for severe hemophilia A, BMN 270 show its potential to benefit patients with unmet medical needs based on early clinical data.

EMA’s committee responsible for human medicines, determined to provide access to the PRIME scheme based on findings that it provides certain unmet needs in prophylactic treatment settings, including hemophilic arthropathy, and that it reduced annulized bleeding rates with patients affected with hemophilia A.

Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin, said. “We look forward to preparing to enroll patients in a registration enbabling study in the third quarter of this year, as we work closely with the EMA to accelerate development and hopefully to facilitate earlier access to the first gene therapy treatment for patients with severe hemophilia A, the most common form of the disease.”

PRIME is a scheme that make medicines considered priority within the European Union (EU).

Through PRIME, EMA offers larger support to drugmakers to optimize the generation of robust data on the drug’s benefits and risks and enable quicker estimation of drug applications.