CrystalGenomics' anticancer drug granted FDA's orphan drug status

South Korea’s CrystalGenomics said Monday that the U.S. Food and Drug Administration (FDA) has designated its anticancer candidate Ivaltinostat as an orphan drug for acute myeloid leukemia (AML).

Ivaltinostat is a novel oncology drug candidate engineered 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) which can cause cancer growth.

With the FDA’s orphan drug designation (ODD), CrystalGenomics will be eligible to be exempt from fees for applying for U.S. regulatory approval and 50 percent tax credit for clinical trial costs.

CrystalGenomics said it plans to expand indications of Ivaltinostat for different cancer types as monotherapy or in combination. It is preparing to conduct a phase 1/2a trial of the drug in combination with an immune checkpoint inhibitor in patients with liver cancer in the U.S.

In the U.S., 19,940 people were diagnosed with acute myeloid leukemia in 2020 alone and 11,180 people died from this disease, according to estimates out from the American Cancer Society. The AML treatment market is projected to be worth $950 million by 2024 in seven major countries.

The Kosdaq-listed stock closed Monday down 1.19 percent at 16,550 won ($15.06) after going as high as 17,300 won.

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