Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Irish firm Kamada has received orphan drug designation from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development for its human Alpha-1 Antitrypsin (AAT) Glassia, to treat immunologically-based graft versus host disease (GVHD).
Subscribe to our email newsletter
According to the preliminary human and animal studies, Glassia may be able to treat and reduce the severity of GVHD, a life threatening complication of allogeneic stem cell transplantation.
GVHD 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.
Glassia is currently used in a Phase I/II clinical trial that is being carried out by the Fred Hutchinson Cancer Research Center in Seattle, Washington, in cooperation with Baxter International and Kamada.
About 24 GVHD patients with inadequate response to steroid treatment following allogeneic bone-marrow stem cell transplant, are being evaluated in the trial.
The patients are enrolled into four dose cohorts, in which they receive up to eight doses of Glassia and the company intends to report interim data from the trial by the end of 2014.
Kamada co-founder and chief executive officer David Tsur said: "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."