The company is currently assessing ALXN1007 in patients with acute GVHD of the lower GI tract, a severe and life-threatening rare autoimmune disorder which can occur as a complication of stem cell or bone marrow transplantation.
The orphan medicinal product status designation offers Alexion with various benefits and incentives in the EU, including a 10-year period of market exclusivity for the indication, if approved.
Alexion executive vice president and global head of R&D Martin Mackay said: “Acute GI-GVHD is a devastating disease in which about one-third of severely affected patients die within the first six months following transplant despite best available care.
“We are pleased that ALXN1007 has received orphan drug designation in the EU, recognizing the urgent need for an effective treatment for GVHD and the potential of ALXN1007 to improve response rates in patients facing this severe and life-threatening disorder.”
GI-GVHD is an immune-mediated disease that affects about 10% of patients who receive an allogeneic hematopoietic stem cell transplant or bone marrow transplant.
Patients with severe, acute GI-GVHD have a 30% to 40% mortality rate within the initial six months post-transplant. Currently, there are limited treatment options in GI-GVHD.