Baxalta Incorporated, a global biopharmaceutical leader, has submitted an application to the US Food and Drug Administration (FDA) for approval of an investigational 20 per cent concentration immunoglobulin subcutaneous (IGSC, 20 per cent) 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).
“Our IGSC, 20 per cent investigational treatment was developed to give patients more IG treatment options that meet their individual needs,” said John Orloff, M.D., executive vice president, head of research & development and chief scientific officer, Baxalta.
“Regulatory reviews are now underway in both Europe and the US, and we look forward to introducing this new IG treatment option as we continue to build on our leadership in the PI community.”
The US filing is based on positive results of a phase 2/3 study of IGSC, 20 per cent in North American patients at least two years old with PI. In the study, IGSC, 20 per cent met the primary endpoint as measured by the rate of acute serious bacterial infections (ASBIs). A single ASBI was reported with IGSC 20 percent treatment, 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 per cent) were completed without a reduction, interruption, or discontinuation for tolerability reasons and 84.9 per cent of infusions were administered using = 2 infusion sites. The rate of local adverse reactions (ARs) to IGSC, 20 per centwas 0.022/ infusion. All (100 percent) of the local ARs were either mild (92.5 per cent) or moderate (7.5 per cent) 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.
Baxalta’s broad portfolio of immunoglobulin treatments aims to offer PI patients worldwide a range of options to meet their individual needs. Baxalta submitted the IGSC, 20 per cent treatment for European review in the second quarter of 2015 and expects to initiate additional global regulatory submissions in 2016.
Primary immunodeficiencies (PI) are a group of more than 200 disorders in which part of the body's immune system is missing or does not function properly. Normally, the immune system protects the body from pathogenic microorganisms like bacteria, viruses, and fungi, which can cause infectious diseases. When any part of a person's immune system is absent or dysfunctional, they are susceptible to infections and may take longer to recover from infections. When a defect in the immune system is inherited, it is called primary immune deficiency. It is estimated that as many as six million children and adults may be affected by PI worldwide.