Twice-Weekly BAX 855 Treatment Viable for Hemophilia

Hemophilia Reports, March 2016, Volume 3, Issue 1

The use of twice-weekly prophylactic regimens to treat hemophilia A was shown to be successful among patients treated with BAX 855.

The use of twice-weekly prophylactic regimens to treat hemophilia A was shown to be successful among patients treated with BAX 855, based on Advate, according to findings released by Baxalta.

The study, published in the journal Blood, built upon data originally presented in 2014. BAX 855, known in the United States as Adynovate, was assessed for safety and efficacy profiles for bleed prevention with a twice-weekly dosing schedule. The mean half-life extension increased between 1.4 and 1.5 fold compared to Advate.

“ADYNOVATE has the potential to offer an important new option for patients, providing the combination of bleed prevention with a simple, twice-weekly dosing schedule,” Leonard Valentino, MD, global head, Hematology Medical Affairs, Baxalta, said in a press release. “The results of this pivotal trial demonstrate the ability of ADYNOVATE to help further personalize treatment regimens to minimize the impact that hemophilia has on patients’ lives.”

A total of 137 patients aged 12 to 65 years were assigned to the twice-weekly prophylaxis group or on demand treatment. The twice-weekly group experienced a 95 percent reduction in a median annualized bleed rate compared to the on-demand treatment arm of the program (1.9 vs. 41.5, respectively). Additionally, BAX 855 was successful in treated all bleeding episodes — 95.9 percent of which were controlled with either one or two infusions.

Nearly all (96.1 percent) of the treatment episodes were rated excellent or good. Almost 40 percent of the prophylactic group experienced no bleeds. Reported side effects included headache, diarrhea, nausea, and flushing in seven patients, though no serious adverse events were reported.

“Upon approval, Adynovate will be one of the first new treatments that we bring to market as Baxalta, representing a major milestone toward our plan to achieve 20 new product launches by 2020,” Ludwig Hantson, chief executive officer and president, Baxalta, added in the statement. “With a decades-long heritage in hematology and commitment to innovation, we continue to build a patient-centric portfolio that provides new solutions for evolving patient needs.”

Studies of BAX 855 continue, the statement explained, especially for children under the age of 12 years with severe hemophilia A. BAX 855 is based on Advate, a drug that has been on the market for more than a decade. It is designed to prolong the amount of FVIII available for the body to use and the technology behind the drug is present in several treatments approved for chronic or otherwise serious conditions.