Kamada Ltd. (TASE: KMDA) has been awarded orphan drug status from the US Food and Drug Administration (FDA) for Glassia to treat Graft-versus-host-disease (GVHD). Orphan drug designation carries multiple benefits, including the availability of grant money, certain tax credits and seven years of market exclusivity, as well as the possibility of an expedited regulatory process.
Preliminary human and animal studies indicate that Glassia may be able to treat and reduce the severity of GVHD, which is one of the key, life threatening complications of allogeneic stem cell transplantation. GVHD is an immunologically-based disease that may result in significant damage to the recipients’ health including damage to multiple organs and tissues such as the liver, gastrointestinal tract, skin and mucosal membranes. Tissue destruction also leads to increased inflammatory signals, perpetuating and augmenting the disease process by contributing to the cytokine storm that fuels GVHD even further and, thereby, the damage continues and its intensity is increased.
In recent years, AAT has been investigated extensively and found to have anti-inflammatory, tissue protective, immune-modulatory and anti-apoptotic properties in direct or indirect consequence of its underlying anti-protease capabilities. These properties may attenuate inflammation by lowering levels of pro-inflammatory mediators such as cytokines, chemokines and proteases that are associated with this severe disease.
Currently, Glassia is being used in a Phase 1/2 clinical study that is being conducted by the Fred Hutchinson Cancer Research Center in Seattle, Washington in cooperation with Baxter International Inc. and Kamada. The Phase 1/2 study is evaluating 24 GVHD patients with inadequate response to steroid treatment following allogeneic bone-marrow stem cell transplant. The patients are enrolled into 4 dose cohorts, in which they receive up to 8 doses of Glassia. Interim data from this study is expected by the end of this year.
Kamada Founder and CEO David Tsur said, “We are pleased with the receipt of orphan drug designation for Glassia to treat GVHD as it is a key milestone that supports our broader regulatory and development strategy. Results from this Phase 1/2 study in GVHD may support global clinical development activities and may serve as a platform to apply for an expansion of the AAT indications to include general organ transplantation, based on a similar mechanism of action. GVHD is a disease of significant unmet medical need and both the disease and current therapy options carry considerable side effects.”
He added, “Given the favorable safety profile of Glassia, there is a strong rationale to support the development of this new indication and an increased likelihood of it becoming an effective therapy for this potentially life threatening disease. We will pursue discussion with the U.S. and European regulators with regard to our development pathway and with an aim to move forward with a more advanced study of Glassia to treat GVHD.”
Published by Globes [online], Israel business news - www.globes-online.com - on October 29, 2014
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