Melbourne, Sep 17, 2008 AEST (ABN Newswire) - Australia's regenerative medicine company, Mesoblast Limited (ASX:MSB)(PINK:MBLTY), today announced that the United States Food and Drug Administration (FDA) has granted an orphan drug designation for the use of the patented adult stem cell technology in patients undergoing bone marrow transplantation.
The FDA awarded Mesoblast's US-based sister company, Angioblast Systems Inc., the right to use the proprietary "off-the-shelf" allogeneic mesenchymal precursor cells for insufficient haematopoietic stem cell production in patients with hematologic malignancies who have failed treatment with conventional chemotherapy.
According to the March issue of Biology and Bone Marrow Transplantation, the probability that an individual in the United States will require a haematopoietic stem cell bone marrow transplant sometime during their life is 1 in 217. The FDA's orphan drug designation is reserved for new drugs or therapies being developed to treat diseases or conditions affecting less than 200,000 patients annually in the United States. Orphan drug designation allows for an accelerated review process by the FDA, seven-year market exclusivity in the United States upon obtaining marketing authorisation, tax benefits, and exemption from user fees.
Hematopoietic stem cells are used to regenerate bone marrow in patients whose own bone marrow is damaged and destroyed by treatments for various cancers. The greater the number of haematopoietic stem cells transplanted, the greater the likelihood that the bone marrow transplant will successfully engraft and regenerate a patient's damaged bone marrow.
In preclinical studies, the patented allogeneic mesenchymal precursor cells have been shown to significantly expand the number of haematopoietic stem cells in culture. The results of these studies formed the basis for the successful orphan drug submission to the FDA.
Mesoblast's Executive Director, Professor Silviu Itescu, said the orphan drug designation was a significant milestone for the platform stem cell technology.
"It broadens the potential commercial applications to conditions requiring repair and regeneration of bone marrow, including various cancers and genetic diseases," he said.
"Significantly, orphan drug designation for our cells in these conditions may facilitate earlier revenues and market exclusivity," Professor Itescu added.
Contact
Julie Meldrum
Corporate Communications Director
Mesoblast Limited
T: + 61 (03) 9639 6036
E: julie.meldrum@mesoblast.com
W: www.mesoblast.com
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