German pharma Bayer has sent its BLA to the FDA for its long-acting Hemophilia A medication BAY94-9027.
The drug, which is a site-specifically PEGylated recombinant human Factor VIII, is hoping to build on last year’s EU approval for Kovaltry (octocog alfa; BAY 81-8973), a hemophilia treatment that expanded the company's already big-selling Kogenate franchise.
Kovaltry is a new plasma-free recombinant Factor VIII product that does not use components of animal or human origin. It was approved for patients of all ages with hemophilia A, and works when used prophylactically either two or three times per week.
Kogenate, meanwhile, is given three times a week, and Bayer hopes that with BAY94-9027, it could be given less than this: In its submission the U.S. regulator, it used data out of the Protect VIII trial, where its medication showed protection from bleeds with dosing intervals when used prophylactically once every week, once every five days, and twice per week.
The idea, of course, is to incrementally lower the rates of treatment as much as possible, an attractive option for those with a bleeding disorder, and the future could come from gene therapy, which is hoping to try and remove the need for frequent dosing.