If at first you don’t succeed, try again. Baxter Healthcare (BAX) and Halozyme Therapeutics, Inc. (HALO) submitted an amended biologics license application (BLA) to the U.S. FDA to restart the review process for HyQvia, an infusion for the treatment of adult patients with primary immunodeficiency (PI).
Primary immunodeficiencies are disorders in which part of the body’s immune system is missing or does not function properly. PI is usually genetic and is not caused by other diseases, drugs or toxic exposure. About 1 in 500 people are born with a primary immunodeficiency, for an estimated six million patients worldwide.
HyQvia is a combination of human immune globulin (IG) and recombinant human hyaluronidase. The drug uses HALO’s Enhanze technology that allows the drugs to be absorbed more quickly, enabling delivery of a full dose of immunoglobulin subcutaneously (by injection) in a single site every three to four weeks. Current treatment regimens require an IV treatment every few weeks or an injection once or twice weekly. The drug was approved in Europe in May of 2013; however, the companies received a complete response letter from the U.S. FDA in April 2012.
The original BLA for HyQvia was based on results from a Phase III, prospective, open-label, non-controlled multi-center clinical trial evaluating the safety and effectiveness of HyQvia in the prevention of acute serious bacterial infections, rate of adverse reactions and the pharmacokinetic parameters compared to immunoglobulin administered intravenously.
While the companies did not make their complete response letter public, they noted that the FDA requested additional preclinical data to support the BLA. In particular, the FDA sought additional data concerning the possible effects non-neutralizing antibodies generated against recombinant human hyaluronidase antibodies on reproduction, development and fertility.
BAX and HALO submitted additional preclinical data that was requested by the FDA in 2012, and expect a six-month review period.
