EU grants orphan drug designation to ENMD-2076 for HCC

CASI Pharmaceuticals, Inc. announced the European Union granted Orphan Drug Designation of an investigational oncology drug, ENMD-2076, for the treatment of hepatocellular carcinoma and fibrolamellar carcinoma, a rare type of HCC.

With the designation status, CASI will be able to market ENMD-2076, an Aurora A/angiogenic kinase inhibitor, exclusively for 10 years in the E.U. after approval, according to the release. The drug is also under investigation for fibrolamellar carcinoma (FLC)

“We are very pleased with the orphan drug designation in the E.U. for HCC,” Ken Ren, PhD, CEO of CASI Pharmaceuticals, said in the release. “We are in the process of initiating our phase 2 clinical trials in the U.S. for the treatment of FLC and expecting to start patient recruitment very soon.”

The FDA granted orphan drug designation for ENMD-2076 to treat HCC in July 2014, which enables CASI to exclusively market the drug for 7 years after approval.

Ren further stated in the release: “We will seek regulatory approvals and expand the trial into E.U. once a signal of clinical benefits is indicated in our U.S. trial. As a part of our global development plan, we also have clinical trial application filed with Chinese FDA pending for approval for FLC. We are quite optimistic and committed to the development of ENMD-2076 as a first line therapy for the treatment of FLC and look forward to advancing its development to the next step.”

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

Disclosures: Ren reports being employed by CASI Pharmaceuticals.