BerGenBio's AML Drug Granted FDA Orphan Status
Oncology biopharmaceutical company BerGenBio announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its investigational drug BGB324 as treatment for acute myeloid leukemia (AML).
BGB324 is a first-in-class, highly selective small molecule that inhibits the Axl receptor tyrosine kinase. The drug blocks the epithelial-mesenchymal transition (EMT), which plays a critical part in building drug resistance and enabling metastasis.
Acute myeloid leukemia is an aggressive cancer that attacks the blood and bone marrow. AML is marked by the excessive growth of abnormal white blood cells, which accumulate in the bone marrow and hinder production of healthy blood cells. AML affects approximately 1 in 227 men and 1 in 278 women. The disease’s five year survival in the U.S. is only 24 percent.
The FDA’s orphan designation is granted to development stage drugs that address rare diseases and conditions that affect less than 200,000 in the U.S. The designation gives sponsor companies several development and commercial incentives including tax credits for qualified clinical testing.
Richard Godfrey, CEO of BerGenBio, said, “The FDA's decision to grant BerGenBio orphan-drug status for BGB324 is a significant milestone for the Company and recognizes the need for innovative new ways to treat AML. The designation will give BerGenBio access to various development incentives from the Agency… and, if the drug receives marketing approval, it will enjoy seven years of market exclusivity in the United States.”
Earlier this month, the company reported the dosing of the first patient in the Phase 1B trial of the drug in AML. The trial will be divided into two parts and will assess the safety and tolerability of BGB324 as a monotherapy and in combination with cytarabine, a standard-of-care therapy. The company said it is preparing to conduct more trials for BGB324 as treatment for non-small cell lung cancer (NSCLC).