The FDA granted orphan drug designation to a liquid alpha-1 proteinase inhibitor for the treatment of patients with graft-versus-host disease following allogeneic bone marrow stem cell transplant, its manufacturer announced recently.
Based on preclinical research, the alpha-1 proteinase inhibitor (Glassia, Kamada Ltd.) — currently indicated as a maintenance therapy in adults with clinically evident emphysema due to congenital alpha-1 antitrypsin deficiency — may aid in reducing inflammation associated with graft-versus-host disease (GVHD).
“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 [alpha-1 antitrypsin] indications to include general organ transplantation, based on a similar mechanism of action,” David Tsur, co-founder and CEO of Kamada, said in a press release. “GVHD is a disease of significant unmet medical need and both the disease and current therapy options carry considerable side effects.”
The FDA based its decision in part on preliminary data from phase 1/2 clinical study evaluating 24 patients with GVHD who exhibited an inadequate response to steroid treatment following allogeneic bone marrow stem cell transplant. The patients were enrolled into four-dose cohorts, in which they received up to eight doses of the proteinase inhibitor.
“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,” Tsur said in the release. “We will pursue discussion with the US 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.”
The FDA Office of Orphan Products grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.