Baxalta files at FDA for approval of Gammagard 20% subcutaneous treament for primary immunodeficiencies

Baxalta announced that it has submitted an application to the FDA for approval of an investigational 20% concentration immunoglobulin subcutaneous (IGSC 20%) treatment for patients with primary immunodeficiencies (PI, a group of disorders in which part of the body's immune system is missing or does not function properly).

The U.S. filing is based on positive results of a Phase II/III study of IGSC 20% in North American patients at least two years old with PI. In the study, IGSC 20% met the primary endpoint as measured by the rate of acute serious bacterial infections (ASBIs). A single ASBI was reported with IGSC 20%, equating to 0.012 ASBI/patient-year (p<0.0001, compared with the reference threshold of one ASBI/patient-year). The rate of all infections was 2.41/patient-year. Nearly all infusions (99.8 percent) were completed without a reduction, interruption, or discontinuation for tolerability reasons and 84.9 percent of infusions were administered using ≤ 2 infusion sites. The rate of local adverse reactions (ARs) to IGSC 20% was 0.022/infusion. All of the local ARs were either mild (92.5%) or moderate (7.5%) in severity. The most common ARs were local adverse reactions, systemic adverse reactions including headache, nausea, fatigue, diarrhea, and vomiting. The complete data will be presented at an upcoming medical congress.