HCC inhibitor receives FDA orphan drug designation

Casi Pharmaceuticals has received an orphan drug designation from the FDA for ENMD-2076 to treat hepatocellular carcinoma, according to a press release.

With the status, Casi will be able to market ENMD-2076, an Aurora A/angiogenic kinase inhibitor, exclusively for 7 years, seek opportunities for additional funding and receive expert protocol assistance, according to the release.

“We are pleased with the orphan drug designation, as it confirms our belief in the versatility of ENMD-2076 as a promising treatment for HCC and for other tumor types that we are currently evaluating in the clinic,” Ken K. Ren, PhD, chief executive officer of Casi Pharmaceuticals, said in the release. “Orphan drug status also enhances the commercial value of ENMD-2076 to treat HCC, a disease which is difficult to treat. We are finalizing our next steps for ENMD-2076 in HCC and/or in fibrolamellar carcinoma, a subset of HCC and for which there is no treatment available, and look forward to advancing our overall development plan for ENMD-2076.”

ENMD-2076 currently is being used in phase 2 clinical trials to treat ovarian cancer, advanced/metastatic soft tissue sarcoma and advanced ovarian clear cell carcinomas, as well as a study for triple-negative breast cancer, the release said.

The FDA already has given orphan drug status to ENMD-2076 to treat ovarian cancer, acute myeloid leukemia and multiple myeloma.