SillaJen's BAL0891 designated an orphan drug by the FDA, boosting hopes for AML drug development

[Financial News] SillaJen's next-generation anticancer drug candidate BAL0891 has been designated as an orphan drug for acute myeloid leukemia (AML) by the U.S. Food and Drug Administration (FDA). SillaJen said on the 25th that BAL0891 had received orphan drug designation from the FDA for the AML indication.
BAL0891 is a first-in-class anticancer candidate that simultaneously targets TTK and PLK1, two key proteins involved in cancer cell division. It is currently undergoing global Phase 1 trials in solid tumors and blood cancers.
FDA orphan drug designation is a system designed to support the development of treatments for rare diseases affecting fewer than 200,000 patients in the United States. With this designation, SillaJen has become eligible to apply for FDA clinical research funding support during BAL0891's development.
The designation also provides benefits such as a 25% tax credit on clinical trial costs in the United States, FDA consultation on clinical trial plans and fast-track review support, and exemption from new drug application fees. If the drug is eventually approved, it could also secure seven years of market exclusivity in the U.S. for the same drug and indication.
AML, the disease targeted by this designation, is a blood cancer in which white blood cells in the bone marrow turn cancerous and multiply rapidly. Because the disease progresses quickly, existing treatments still face limitations such as side effects, relapse, and drug resistance.
As BAL0891 simultaneously targets TTK and PLK1, which are key factors in the cancer cell division process, it is drawing attention for its potential to become a new treatment option for AML patients who have developed resistance to existing therapies.
A SillaJen official said, "Being designated as an orphan drug at such an early stage of clinical development suggests that BAL0891's medical significance and potential have been recognized to some extent." The official added, "Based on this achievement, we will do our best to deliver even more positive results in the remaining clinical trials."

[email protected] Kang Jung-mo Reporter