BioMarin Wins Award for Getting Gene Therapy Facility Operational Ahead of Time

BioMarin Pharmaceutical has won an award for getting its new gene therapy facility in Novato, California, operational ahead of schedule.

The International Society for Pharmaceutical Engineering (ISPE) award was for project execution. It was part of the society’s 2018 Facility of the Year honors.

BioMarin took only a year from the time it began the work to complete one of the first gene therapy manufacturing facilities of its kind in the world.

The company’s lead gene therapy candidate is a hemophilia A treatment that it calls valoctocogene roxaparvovec (BMN 270).

Hemophilia A is caused by a deficiency of clotting factor VIII, a protein that works with blood platelets in the clotting process. When blood vessels are injured, clotting factors help platelets stick together to plug cuts and breaks on the vessels, stopping the bleeding.

In mouse models of the disease, valoctocogene roxaparvovec restored factor VIII plasma concentrations to levels normally seen in healthy humans, the company has reported.

The gene therapy is designed to deliver the missing gene needed to produce factor VIII in a single administration. If it proves effective during the clinical trials process, it would essentially mean a cure for hemophilia A, eliminating the need for ongoing treatments of factor VIII.

Valoctocogene roxaparvovec has advanced to the Phase 3 stage of the trials.

An urgent need to produce material for the trials prompted BioMarin to convert an office and warehouse building into an 18,000-square-foot gene therapy facility. It will allow the company to proceed with its development program with no hiccup.

The initial goal was to release the first filled vials of the gene therapy for Phase 3 trials less than 15 months from the start of the construction project. Beating the goal, the buildout and commissioning of the facility was accomplished in less than a year.

“The buildout of our gene therapy manufacturing facility was a logistical challenge that required leveraging our experience gained during licensure of our Galli and Shanbally biological facilities,” Robert Baffi, BioMarin’s executive vice president of technical operations, said in a press release. “This plant provides us with scheduling flexibility, allowing for rapid product development, and is capable of providing sufficient capacity to meet clinical and projected commercial requirements.”

The award was announced on March 20, during the 2018 ISPE Europe Annual Conference in Rome.

BioMarin’s global Phase 3 program for valoctocogene roxaparvovec includes two trials. One, GENEr8-1, involes a 6e13 vg/kg dose (NCT03370913). The other, GENEr8-2, involves a 4e13 vg/kg dose. It is not registered yet, so at the moment there is no trial number.

The two trials will evaluate valoctocogene roxaparvovec’s safety and effectiveness. The primary goal will be to see how much factor VIII the replacement gene can generate. A secondary goals will be to see how the therapy affects patients’ annualized bleed rate.

BioMarin announced dosing of the first patient in GENEr8-1 in December 2017. It expects to enroll the first patient in the GENEr8-2 trial soon.