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FDA grants orphan drug designation to BI 836858 for myelodysplastic syndrome
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The FDA granted orphan drug designation to BI 836858 for the treatment of myelodysplastic syndrome, according to the drug’s manufacturer.
BI 836858 (Boehringer Ingelheim) is an anti-CD33 monoclonal antibody.
“Preclinical studies ... have shown promising therapeutic potential in myelodysplastic syndrome (MDS), a rare group of bone marrow disorders,” Martina Flammer, MD, vice president of clinical development and medical affairs specialty care at Boehringer Ingelheim, said in a company-issued press release. “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 1/phase 2, multicenter, open-label, dose-escalating randomized trial designed to evaluate BI 836858 in patients with MDS is underway.
The FDA previously granted orphan drug designation to BI 836858 for the treatment of patients with acute myeloid leukemia.
BI 836858 also is being evaluated as part of the Leukemia & Lymphoma Society–led Beat AML Master Trial, designed to match newly diagnosed patients aged 60 years or older with the most appropriate investigational drug or drugs that can attack the genetic mutations detected in their cancers.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.