BI 836858 was previously granted orphan drug designation for the treatment of patients with AML.

FDA has granted orphan drug designation to Boehringer Ingelheim's anti-CD33 monoclonal antibody BI 836858 for the treatment of myelodysplastic syndromes (MDS).

MDS are a group of bone marrow disorders. In MDS, the bone marrow is characterized by cell abnormalities and ineffective blood cell production. Various subtypes of the disease exist with variable prognoses and treatment options. In some cases, MDS can progress to acute myeloid leukemia (AML), an aggressive and devastating blood cancer. According to data from the National Cancer Institute, an estimated 15,000 new cases of MDS are identified each year.

"Preclinical studies with our anti-CD33 monoclonal antibody have shown promising therapeutic potential in myelodysplastic syndromes, a rare group of bone marrow disorders," said Martina Flammer, Vice President, Clinical Development & Medical Affairs Specialty Care, Boehringer Ingelheim. "Orphan drug designation for our investigational candidate in MDS is an important recognition of our ongoing research efforts and underscores Boehringer Ingelheim's commitment to studying treatment options for rare cancers."

A Phase I/II multi-center, open-label, dose escalation and randomized trial (NCT02240706) evaluating BI 836858 in patients with MDS is ongoing. BI 836858 previously received orphan drug designation for the treatment of patients with AML and is currently being evaluated as part of the Leukemia & Lymphoma Society's (LLS) groundbreaking, first-of-its-kind Beat AML Master trial program to advance treatment for patients with AML. Using the latest genomic technology, the trial finds and matches specific AML mutations in newly-diagnosed patients over the age of 60 with an investigational drug or drugs best suited to attack the specific genetic mutations found within the cancer.

BI 836858 is a monoclonal antibody that targets a protein called CD33, which is expressed on the surface of certain cancerous cells. BI 836858 has been engineered for improved antibody-dependent cell-mediated cytotoxicity (ADCC), a type of immune reaction in which a target cell or microbe is coated with antibodies and killed by certain types of white blood cells. Preclinical studies with the compound have shown promising ADCC against malignant cells.