Kiadis Pharma announced that its lead product ATIR has been granted orphan drug designation (ODD) by the European Medicines Agency (EMEA) for the prevention of acute Graft versus Host Disease (GvHD) following an allogeneic bone marrow transplantation.

“Following the orphan drug designation granted by the FDA for our lead product ATIR, this is another important milestone in the development of ATIR as a novel approach which may enable a safe and potentially life-saving mismatched bone marrow transplantation as a treatment option for end-stage blood cancer patients” says Dr. Manja Bouman, chief executive officer of Kiadis Pharma.

The EMEA’s orphan drug designation is reserved for new therapies being developed to treat life-threatening or chronically debilitating diseases or conditions that are relatively rare in the European Union and for which no satisfactory therapy is available. The orphan drug designation provides for incentives to support research and development, exemption from user fees and a ten-year period of market exclusivity in the European Union after product approval.

ATIR is under development to allow early immune reconstitution while preventing life-threatening GvHD, a major complication of allogeneic bone marrow transplantations. GvHD is a condition caused by the donor immune cells attacking the patient’s tissues and organs. Allogeneic bone marrow transplantations today require that the patient and donor immune systems are highly similar in order to reduce the risk of GvHD and therefore these transplantations rely on matching donors. By eliminating those immune cells which could attack the patient’s body and thus preventing the occurrence of GvHD, ATIR enables the use of a mismatched donor. This means ATIR will address a significant limitation in bone marrow transplantations, which is the timely availability of a donor.

Every year more than 70,000 patients die of blood cancers in the European Union alone. For these end-stage and high-risk blood cancer patients, a bone marrow transplantation might be the only treatment option which can lead to prolonged survival or cure. Annually about 9,000 patients in the European Union receive a bone marrow transplant from a matched donor. However, this is only 1/3 of the patients in the European Union who are in need of such a transplant, leaving the majority of patients without the option of a potential curative therapy