Boehringer Ingelheim has bagged US orphan drug designation for its anti-CD33 monoclonal antibody BI 836858 for the treatment of myelodysplastic syndromes (MDS).

MDS are a group of bone marrow disorders, in which the bone marrow carries cell abnormalities and is unable to produce blood cells efficient. There are many subtypes of the disease - including the blood cancer acute myeloid leukaemia (AML) - all with variable prognoses and treatment options.

BI 836858 is a monoclonal antibody that targets a protein called CD33, which is expressed on the surface of certain cancerous cells.

The drug 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.

According to the German drugmaker, preclinical studies with the compound have shown promising ADCC against malignant cells, and a Phase I/II multi-center, open-label, dose escalation and randomised trial evaluating BI 836858 in patients with MDS is ongoing.

The drug is also currently being evaluated as part of the Leukaemia & Lymphoma Society's (LLS) first-of-its-kind Beat AML Master trial programme to advance treatment for patients with AML, which uses genomic technology to find 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.