BANNOCKBURN, Ill.--(BUSINESS WIRE)--Baxalta Incorporated (NYSE:BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced that it has submitted an application to the U.S. Food and Drug Administration (FDA) for approval of an investigational 20 percent 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 properly1).
"Our IGSC, 20% 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 U.S., and we look forward to introducing this new IG treatment option as we continue to build on our leadership in the PI community."
The U.S. filing is based on positive results of a Phase 2/3 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 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 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 (100 percent) of the local ARs were either mild (92.5 percent) or moderate (7.5 percent) 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% treatment for European review in the second quarter of 2015 and expects to initiate additional global regulatory submissions in 2016.
About Primary Immunodeficiency
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.2 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.3 It is estimated that as many as six million children and adults may be affected by PI worldwide.1
Baxalta Incorporated (NYSE: BXLT) is a $6 billion global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in hematology, oncology and immunology. Driven by passion to make a meaningful impact on patients' lives, Baxalta's broad and diverse pipeline includes biologics with novel mechanisms and advanced technology platforms such as gene therapy. The Baxalta Global Innovation and R&D Center is located in Cambridge, Massachusetts. Launched in 2015, following separation from Baxter International Inc, Baxalta's heritage in biopharmaceuticals spans decades. Baxalta's therapies are available in more than 100 countries and it has advanced biological manufacturing operations across 12 facilities, including state-of-the-art recombinant production and plasma fractionation. Headquartered in Northern Illinois, Baxalta employs 16,000 employees worldwide.
This release includes forward-looking statements concerning Baxalta's IGSC 20 percent treatment, including expectations with regard to future regulatory actions and potential impact on patients. Such statements are made of the date that they were first issued and are based on current expectations, beliefs and assumptions of management. Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond Baxalta's control and which could cause actual results to differ materially from those in the forward-looking statements, including the following: clinical trial results; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; product quality, manufacturing or supply issues; patient safety issues; and other risks identified in Baxalta's Registration Statement on Form 10 and other Securities and Exchange Commission filings, all of which are available on Baxalta's website. Baxalta expressly disclaims any intent or obligation to update these forward-looking statements except as required by law.
1. Bousfiha AA et al. Primary immunodeficiency diseases worldwide: more common than generally thought. J Clin Immunol. 2013 Jan;33(1):1-7.
2. Al-Herz, Waleed, et al. Primary immunodeficiency diseases: an update on the classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency. Frontiers in Immunology 2014; V5:162: 1-33.
3. IDF Patient & Family Handbook for Primary Immunodeficiency Diseases. 5th edition. Blaese. 2013.