[E-Daily Reporter NA EUN-KYUNG ] SillaJen ( SillaJen, Inc.(215600)) announced on the 25th that its next-generation anticancer candidate “BAL0891” has been designated as an orphan drug by the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML).

With this designation, SillaJen, Inc. can now utilize the FDA’s Orphan Drug Program during the development of BAL0891 for AML. The company is eligible for benefits such as funding for clinical research, tax credits for clinical trial costs in the U.S., FDA consultation on clinical trial plans, and waivers of new drug application fees. If the company successfully obtains final marketing approval, it will, in principle, be granted a seven-year market exclusivity period in the U.S. for the same drug and indication.

The FDA’s Orphan Drug Designation is a program intended to support the development of treatments for rare diseases affecting fewer than 200,000 patients in the United States. However, the designation itself does not imply recognition of the candidate drug’s efficacy, safety, or likelihood of future marketing approval.

BAL0891 is an anticancer candidate designed to simultaneously inhibit threonine/tyrosine kinase (TTK) and polo-like kinase 1 (PLK1), both of which are involved in cancer cell division. SillaJen, Inc. is developing BAL0891 as a “first-in-class” candidate that simultaneously targets TTK and PLK1. A global Phase 1 clinical trial is currently underway for solid tumors and blood cancers.

Acute myeloid leukemia (AML), the condition for which BAL0891 has been designated as an orphan drug, is a blood cancer in which abnormal myeloid cells rapidly proliferate in the bone marrow, interfering with the production of normal blood cells. The disease progresses rapidly, and treatment options and prognosis vary significantly depending on the patient’s age and genetic mutations.

Treatments for acute myeloid leukemia include chemotherapy, hematopoietic stem cell transplantation, and targeted therapies. Although therapies targeting specific genetic mutations have been introduced in recent years, there remains a need for new treatment options due to issues such as relapse and drug resistance.

SillaJen, Inc. plans to leverage this Orphan Drug designation to continue the clinical development of BAL0891 for AML.

A SillaJen, Inc. spokesperson stated, “With BAL0891 receiving FDA orphan drug designation for acute myeloid leukemia, we will now be able to utilize relevant support programs during future clinical trials and the approval process,” adding, “We will proceed with the ongoing clinical development without delay to validate the potential of BAL0891.”