FDA grants orphan drug status to Boehringer’s BI 836858 to treat MDS

Orphan drug designation is granted by the FDA to investigational compounds intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people.

MDS are a group of bone marrow disorders. The bone marrow produces blood cells. 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.

Boehringer Ingelheim Clinical Development & 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."

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.