Cellenkos’ CK0801 Granted FDA Orphan Drug Designation for Aplastic Anemia
Cellenkos® Inc. announced that the FDA has granted Orphan Drug Designation to CK0801, its off-the-shelf, allogeneic T regulatory (Treg) cell therapy, for the treatment of Aplastic Anemia—a rare and life-threatening bone marrow failure disorder affecting fewer than 200,000 individuals in the U.S. The designation provides Cellenkos with regulatory and financial incentives, including potential seven-year market exclusivity upon approval. Aplastic Anemia has a projected U.S. prevalence of about 5,000 patients.
Encouraging data from a Phase I trial (NCT03773393), published in NEJM Evidence, sho...
