FDA Grants Orphan Designation to Bone Marrow Disease Drug

Myelodysplastic syndrome can lead to aggressive leukemia.

Boehringer Ingelheim recently announced the FDA has granted BI 836858 orphan drug designation for the treatment of myelodysplastic syndromes (MDS). The drug is an investigational anti-CD33 monoclonal antibody.

BI 836858 was engineered to improve antibody-dependent cell-mediated cytotoxicity, which is an immune process that coats a cell with an antibody and is killed by white blood cells, according to a press release. Preclinical trials have shown promise against cancer cells.

MDS are a group of rare bone marrow disorders that are characterized by cell abnormalities and poor blood cell production, according to the release. Subtypes of MDS currently have differing prognoses and treatment options. Some forms of MDS can be developed in response to cancer treatment or chemical exposure, while others have unknown origins, according to the Mayo Clinic.

While certain forms can be treated with blood transfusions, drugs, or bone marrow transplants, some patients with MDS can develop acute myeloid leukemia, which is an aggressive form of the disease.

Included in a phase 1/2 dose escalation trial were patients with MDS who were randomized to receive BI 836858. The trial is ongoing, but preliminary and preclinical data are promising in these patients.

The FDA previously granted BI 836858 orphan drug designation for the treatment of acute myeloid leukemia, and is being tested by the Leukemia & Lymphoma Society’s treatment program, according to the press release. In this study, the investigators performed genomic analyses, and matches mutations in newly-diagnosed patients with a drug best suited to target specific mutations.

The latest orphan drug designation granted to BI 836858 will speed the approval process for the treatment of patients with MDS, where there are nearly 15,000 new diagnoses 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, MD, 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."