FDA grants orphan drug designation to Boehringer Ingelheim’s antibody BI 836858


The US Food and Drug Administration (FDA) has granted orphan drug designation to Boehringer Ingelheim’s anti-CD33 monoclonal antibody, BI 836858, which is being investigated in the treatment of myelodysplastic syndromes (MDS).

The monoclonal antibody targets a protein called CD33, which is expressed on the surface of certain cancerous cells and has been engineered for improved antibody-dependent, cell-mediated cytotoxicity (ADCC).

This designation is granted to investigational compounds intended for the safe treatment, diagnosis, or prevention of rare diseases that affect less than 200,000 people.

MDS are a group of bone marrow disorders characterised by cell abnormalities and ineffective blood cell production.

In some cases, MDS can progress to acute myeloid leukaemia (AML), an aggressive and devastating blood cancer.

"This designation is granted to investigational compounds intended for the safe treatment, diagnosis, or prevention of rare diseases that affect less than 200,000 people."

Boehringer Ingelheim clinical development and medical affairs specialty care vice-president Martina Flammer said: "Preclinical studies with our anti-CD33 monoclonal antibody have shown promising therapeutic potential in myelodysplastic syndromes, a rare group of bone marrow disorders.

"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."

The company is presently conducting a Phase I/II multi-centre, open-label, dose escalation and randomised trial (NCT02240706) to evaluate BI 836858 in patients with MDS.

BI 836858 previously received orphan drug designation to treat patients with AML.

It is currently being evaluated as part of the Leukaemia & Lymphoma Society's (LLS) Beat AML Master trial programme to advance treatment for patients with AML.