FDA grants orphan drug designation to BLU-554 for HCC

The FDA granted orphan drug designation to Blueprint Medicines drug candidate BLU-554 for the treatment of hepatocellular carcinoma, according to a press release.

BLU-554 (Blueprint Medicines), an inhibitor of fibroblast growth factor receptor 4 (FGFR4), has been shown to have significant anti-tumor activity in preclinical models of HCC driven by aberrant FGFR4 signaling, according to the release. In an analysis conducted by Blueprint Medicines, the signaling of FGFR4 could be a “key driver in up to 30% of HCC patients.”

“We believe BLU-554 represents a promising new approach for molecularly targeted therapy in HCC,” Jeffrey Albers, CEO of Blueprint Medicines, said in the release. “Patients with advanced HCC have a poor prognosis, and there's a clear need for better treatment options. We are pleased that we continue to make progress toward our goal of delivering a highly targeted medicine to improve the lives of HCC patients.”

The release states BLU-554 is currently being investigated in a phase 1 clinical trial in patients with advanced HCC and cholangiocarcinoma.

The FDA’s 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.

Disclosures: Albers reports being employed by Blueprint Medicines.