UK Regulatory Agency Approves Continued Enrollment in BioMarin Phase 1/2 Study of BMN 270 in Hemophilia A

Phase 1/2 Study Expected to Resume by End of 2016

Requirement for Prophylactic Corticosteroids Removed

SAN RAFAEL, Calif., Oct. 13, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A.  BioMarin had previously announced that after enrolling the first 9 patients in the study, that dosing of patients had been suspended due to observed increases in alanine aminotransferase (ALT) levels that exceeded a pre specified threshold set by the company.  Following study suspension, the company reviewed safety and efficacy data on the 9 patients with the MHRA, and based on its review, the MHRA approved resumption of the study. The agency also approved the company’s proposed amendments to the study, which included eliminating the requirement for prophylactic corticosteroids and increasing potential additional enrollment from up to three additional patients to up to six additional patients.

BioMarin intends to resume enrollment in the Phase I/2 study before the end of 2016.  Based on protocol amendments, three patients will be enrolled at a dose of 4 x 1013 vg/kg, and an additional three may be enrolled at this dose or the previously tested high dose of 6 x 1013 vg/kg.  In the up to six additional patients, the requirement for prophylactic corticosteroids has been removed and the threshold for starting therapeutic corticosteroids has been increased.  Safety and efficacy data from these patients will inform the Phase 2b study planned to begin in the second half of 2017.

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