CrystalGenomics said Monday that the U.S. Food and Drug Administration has designated its anticancer candidate Ivaltinostat as an orphan drug for liver cancer.

Ivaltinostat is a new anticancer candidate with advantages of excellent therapeutic effects, low side-effects, and immune balance inducement as a best-in-class molecular targeted inhibitor that disrupts the function of histone deacetylase (HDAC).

CrystalGenomics’ liver cancer drug candidate Ivaltinostat has been designated as an orphan drug by the U.S. FDA. (CrystalGenomics)

With the FDA’s orphan drug designation (ODD), CrystalGenomics will enjoy the exemption of fees for applying U.S. regulatory approval and 50 percent tax credit for clinical trial costs.

The company can also secure marketing exclusivity for Ivaltinostat targeting liver cancer for up to seven years in the U.S., where generic drugs cannot enter the market after the original drug completes the process of receiving approval.

The company plans to expand indications of Ivaltinostat for different types of cancers and enhance efficiency via combination therapy. It is preparing to conduct phase 1/2a trials of combined administration of Ivaltinostat and immune checkpoint inhibitors on patients with liver cancer in the U.S.

“We may be able to advance the clinical trial and the time of approval thanks to FDA’s designation of Ivaltinostat as an orphan drug for liver cancer,” a CrystalGenomics official said. “We will develop a drug applicable to various cancer types.”

It was the third time that the FDA designated a new drug candidate developed by CrystalGenomics as an orphan drug. Another type of Ivaltinostat being developed as a treatment for pancreatic cancer received ODD in August 2019.

The first candidate, CG-806 for acute myeloid leukemia, won the ODD status in December 2017. Aptose Biosciences, a U.S. biotechnology company, is conducting a clinical study on the drug.

shim531@docdocdoc.co.kr

<© Korea Biomedical Review, All rights reserved.>