August 8, 2017
By Mark Terry, BioSpace.com Breaking News Staff
San Rafael, Calif. – BioMarin Pharmaceutical yesterday celebrated both its 20th anniversary and the opening of a new manufacturing facility in Novato, Calif.
BioMarin focuses on rare diseases. The company expects to use the new facility to manufacture BMN 270, a treatment for severe hemophilia A. In a July statement, Robert Baffi, BioMarin’s executive vice president of Technical Operations, said, “BioMarin has built a manufacturing facility that can produce BMN 270 at the scale and quantity to support clinical development and projected commercial demand, if approved. We drew upon our expertise in manufacturing complex biologics to efficiently build one of the largest gene therapy manufacturing facilities in the world, which will allow us to control scheduling, quality and costs to facilitate rapid product development.”
The company had presented data for BMN 270 at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress in Berlin, Germany in July. In the Phase I/II trial, 15 patients with severe hemophilia A received a single dose of BMN 270. Seven received a dose of 6e13 vg/kg; the other six were given a lower dose of 4e13 vg/kg. Two more patients were given lower doses as part of a dose escalation study, but no therapeutic efficacy was observed.
The seven patients receiving the high dose had an average 129 percent Factor VIII activity level by week 24. By week 52, they had an average Factor VIII activity level of 104 percent, which is considered normal range. The six patients on the lower dose had an average 33 percent Factor VIII activity level by week 24, which is classified as mild range for hemophilia. The company plans to advance the higher dose into a Phase III trial in the fourth quarter of this year.
The European Medicines Agency (EMA) granted Biomarin access to its Priority Medicines (PRIME) regulatory initiative for BMN 270.
“The data continue to build the clinical case for the potentially groundbreaking impact of BMN 270 gene therapy for treating patients with hemophilia A,” said John Pasi, the primary investigator for the Phase I/II clinical trials, in a July statement. Pasi, a researcher at Barts and the London School of Medicine and Dentistry and Hemophilia Clinical Director at Barts Health NHS Trust, added, “In addition to the clinical data showing meaningful improvement in bleeds and Factor VIII levels up to 52 weeks, the quality of life data from the patients at the highest dose demonstrate that the potential clinical benefit could also respresent a tangible improvement in a patient’s quality of life.”
The newly renovated facility in Novato is 25,000 square feet. Hank Fuchs, president of BioMarin’s worldwide research and development, said at the ceremony that the facility is now “the first and largest gene manufacturing facility in the world. The scientific possibilities that come from this building, and the hope they offer to patients with rare diseases, are enormous.”
The facility was completed almost 11 months ahead of schedule.
About 700 people work on the Novato campus, with another 1,000 at the company’s headquarters in San Rafael. It employs 2,200 people globally. BioMarin has six approved drugs on the market with four more in development. Last year it brought in about $1.1 billion in revenue.
The company also has a strong college internship program, where it has trained more than 300 students. It recently announced a teen mentorship program with San Marin High School and Biotech Partners, a nonprofit that helps under-served kids with careers in the life sciences.
