CASI Pharmaceuticals, Inc., a biopharmaceutical company, announced that its oncology drug candidate, ENMD-2076, has received Orphan Drug designation from the European Medicines Agency (EMA) for the treatment of hepatocellular carcinoma (HCC), including fibrolamellar carcinoma (FLC), a rare type of HCC.

The designation provides CASI with 10 years of market exclusivity in EU after ENMD-2076 receives marketing authorisation there. The product was also granted Orphan Drug designation for the treatment of HCC by US Food and Drug Administration (FDA) in 2014, which provides CASI with a 7-year market exclusivity in the country after its New Drug Application approval.

Dr. Ken Ren, chief executive officer of CASI, commented, “We are very pleased with the Orphan Drug designation in the EU for HCC. This is an important regulatory milestone for us to enhance the commercial value of ENMD-2076 for global market. We are in the process of initiating our phase 2 clinical trials in the US for the treatment of FLC and expecting to start patient recruitment very soon. We will seek regulatory approvals and expand the trial into EU once a signal of clinical benefits is indicated in our US 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.”

ENMD-2076 is an orally-active, Aurora A/angiogenic kinase inhibitor with a unique kinase selectivity profile and multiple mechanisms of action. ENMD-2076 has been shown to inhibit a distinct profile of angiogenic tyrosine kinase targets in addition to the Aurora A kinase. Aurora kinases are key regulators of mitosis (cell division), and are often over-expressed in human cancers. ENMD-2076 also targets the VEGFR, Flt-3 and FGFR3 kinases, which have been shown to play important roles in the pathology of several cancers. ENMD-2076 has shown promising activity in phase 1 clinical trials in solid tumours including ovarian, breast, liver, renal and sarcoma, as well as in leukemia and multiple myeloma. ENMD-2076 is currently in phase 2 clinical trials in multiple indications, including triple-negative breast cancer, soft tissue sarcoma, ovarian clear cell carcinomas and fibrolamellar carcinoma. ENMD-2076 has received orphan drug designation from the US FDA for the treatment of ovarian cancer, multiple myeloma, acute myeloid leukemia, and hepatocellular carcinoma.