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Kamada’s GvHD treatment AAT gets European orphan drug status

Israel-based Kamada has received orphan drug designation from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) for its human intrevenous (IV) Alpha-1 Antitrypsin (AAT) to treat graft-versus-host disease (GvHD).

The company’s AAT therapy is being examined in a clinical US Phase I/II trial that is evaluating 24 GvHD patients with inadequate response to steroid treatment following allogeneic bone-marrow stem cell transplant.

In the trial, which is being conducted by the Fred Hutchinson Cancer Research Center in Seattle in cooperation with Baxter International, patients were enrolled into four dose groups to receive up to eight doses of AAT.

Preliminary results from this trial showed that continuous administration of AAT as therapy for steroid-resistant gut GvHD is feasible in the subject population.

Kamada co-founder and chief executive officer David Tsur said: "Receipt of European Orphan Drug designation for our AAT to treat GvHD is a key milestone that supports our global regulatory and development strategy.

"GvHD is a disease of significant unmet medical need, and both the disease and current therapy options carry considerable, debilitating side effects."

In recent years, AAT has been examined 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.

Tsur added: "The positive interim results from ongoing studies are very encouraging and support continuation of our global clinical development plans for AAT in treating and preventing GvHD.

"Importantly, the preclinical data support the positive interim results from the Phase I/II clinical study of AAT, which is aimed at treating the gut involvement in steroid-resistant GvHD.

"Given the favorable safety profile of AAT, there is a strong rationale to support the development of this new indication and an increased likelihood of our AAT becoming an effective therapy for this potentially life-threatening disease."

Based on these results, the company now plans to start a Phase III trial in 2016, in order to bring this treatment to the market not before 2019.