Boehringer Myelodysplastic Syndromes Therapy Given Orphan Status

FDA has granted Boehringer Ingelheim an orphan drug designation for its anti-CD33 monoclonal antibody BI 836858 for treating myelodysplastic syndromes (MDS). “Preclinical studies with our anti-CD33 monoclonal antibody have shown promising therapeutic potential in myelodysplastic syndromes, a rare group of bone marrow disorders,” the company says. A Phase 1-2 multi-center, open-label, dose escalation and randomized trial (NCT02240706) evaluating BI 836858 in patients with MDS is ongoing.

BI 836858 is a monoclonal antibody that targets a protein called CD33, which is expressed on the surface of certain cancerous cells. It has been engineered for improved antibody-dependent cell-mediated cytotoxicity, an immune reaction where a target cell or microbe is coated with antibodies and killed by certain types of white blood cells, according to the company.