Netherlands Kiadis gets EMA orphan drug status for ATIR to treat acute myeloid leukemia

PBR Staff Writer Published 03 November 2014

Dutch biopharmaceutical firm Kiadis Pharma has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for its T-cell immunotherapy based medicinal product ATIR to treat acute myeloid leukemia (AML).

Previously, ATIR was also granted ODD by both the EMA and the US Food and Drug Administration (FDA) for the prevention of acute graft versus host disease (GvHD) following an allogeneic bone marrow transplantation.

ATIR allows stem cell transplantations using partially mismatched family members as donors for patients suffering from blood cancer who do not have a standard of care matching stem cell donor available.

For many patients hematopoietic stem cell transplantation (HSCT) is the only potentially curative option but a matching donor is available for only half of the patients in need.

Kiadis Pharma chief executive officer Manfred Ruediger said: "This second Orphan Drug Designation for our lead product ATIR in the European Union represents another very important milestone in the development of our product.

"This, coupled with the previously granted ODDS for ATIR by the FDA and EMA, further highlights our product's importance as a novel approach which may help provide a potentially life-saving transplant from a family member to those patients who otherwise do not find a matched donor in time.

"In many cases, this will be the only remaining option for these patients.

"This news comes directly after having successfully closed an equity financing round with our existing investors which emphasizes the belief our investors have in both the Company and ATIR."

The ODD was based on data from a Phase I/II trial in which high-risk leukemia patients with very poor prognosis were treated with escalating doses of ATIR after a haploidentical HSCT.

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Netherlands Kiadis gets EMA orphan drug status for ATIR to treat acute myeloid leukemia