Pall Helps BioMarin Produce New Gene Therapy Drug for Hemophilia A

Time is critical for patients with rare diseases like hemophilia A, a genetic bleeding disorder that affects an estimated 1 in 4,000-5,000 male births. That's why BioMarin Pharmaceuticals (San Rafael, CA) strives to quickly develop important therapies for hemophilia A and other diseases driven by genetic causes.

Last year, BioMarin turned to Pall to help produce a gene therapy drug called BMN 270, an investigational drug for the treatment of hemophilia A. To manufacture the drug (named a Priority Medicine by the European Medicines Agency), BioMarin needed to build a new manufacturing facility in Novato, CA, and have it fully operational within 18 months. The timeline was aggressive—less than half the time typically required for a project this size. With a clinical trial set to begin at the end of 2017, BioMarin had to be sure the final drug would be ready in time.

The promise of BMN 270

If successful, BioMarin's newest gene therapy has the promise of delivering, in as little as a single administration, the missing gene needed to produce factor VIII (the clotting factor deficient in hemophilia A patients). This provides a potential cure and may eliminate the need for ongoing treatments, which for some hemophilia A patients, can include intravenous infusions three times a week.

Pall Speeds Time to Market

In January 2016, Pall got to work and proposed Single Use Technology (SUT) to speed implementation. Solutions would include Lev mixer technology, plus turbine mixers, transfer assemblies, filling assemblies and filtration assemblies. By September, Pall received its first capital order (valued at $1.2 million), followed by a second order for single-use tangential flow filtration (TFF) systems (worth $250,000) in December. As BioMarin's confidence in Pall grew, so did the project scope. Eventually, BioMarin also added Powder Transfer Vessel (PTV) solutions and single-use drug product filling solutions to the mix—ultimately purchasing $3.6 million in solutions.

The entire project was a complex endeavor with many individual contributors from Pall's commercial, technical, customer, service, marketing, operations, engineering and quality teams—but well worth it, to help bring an important new drug to market and improve life for Hemophilia A patients.

By July, BioMarin had successfully completed its Process Development run at scale, as well as its first Engineering run, and had begun Validation runs. BioMarin expects to begin actual drug production this month (September).

"This success story really highlights the power of single use technology, which compared to traditional stainless steel factories, helped cut our customer's time to market by half—and meant that therapy solutions could hit the market earlier, giving patients relief," said Jeff Kelly, VP Sales, SUT/Biopharm in the Americas. "The quality support and the drive to meet Biomarin's request dates from Pall associates also made a huge impression on BioMarin, which opens the door for more business in the future. We promised what we could do, then we delivered."  

In the future, BioMarin plans to build a second, larger facility to expand manufacturing for BMN 270, which will likely be another opportunity for Pall.