FDA approves new drug applications for potential HCC, GIST drugs

Blueprint Medicines announced that the FDA has approved two Investigational New Drug applications to begin phase 1 clinical trials for BLU-554 for the treatment of advanced hepatocellular carcinoma and cholangiocarcinoma, and BLU-285 for gastrointestinal stromal tumor, according to a press release from the manufacturer.

BLU-554, a potent and selective inhibitor of fibroblast growth factor receptor 4, will be used in a phase 1 trial with 50 patients with advanced, unresectable hepatocellular carcinoma and 10 patients with advanced, resectable cholangiocarcinoma to test the safety and tolerability of escalating doses, according to the release.

BLU-285, a potent and selective inhibitor of KIT Exon 17 and PDGFR-alpha D842V mutants, will also be used in a phase 1 trial of 60 patients with gastrointestinal stromal tumor or other relapsed or refractory solid tumors and will test the safety and tolerability of escalating doses, according to the release.

The release also stated that the goals of both trials are to test the safety and efficacy of the drugs in an effort to establish a maximum tolerated dose.

“We are delighted to reach this important milestone in the development of our two lead drug candidates to meet the urgent needs of patients facing a poor prognosis and few, if any, viable treatment options,” Jeffrey Albers, CEO of Blueprint Medicines, said in the release. “This achievement is a testament to our team’s ability to execute on our stated goal of simultaneously advancing multiple programs into clinical trials.”

The clinical trials will be held at multiple sites across the U.S., Asia and European Union.

Disclosures: Albers reports being employed by Blueprint Medicines.