SillaJen(215600) said on the 25th that its anticancer candidate "BAL0891" has been designated an orphan drug for acute myeloid leukemia (AML) by the U.S. Food and Drug Administration (FDA).
BAL0891 is a first-in-class anticancer candidate that simultaneously targets TTK and PLK1, key proteins involved in cancer cell division. It is currently in a global phase 1 trial for solid tumors and blood cancers.
The FDA's orphan drug designation is granted to medicines being developed for rare diseases that affect fewer than 200,000 patients in the United States. SillaJen, with this designation, can receive benefits including ▲ eligibility to apply for FDA clinical research funding programs ▲ a 25% tax credit on clinical trial expense in the United States ▲ FDA advice and expedited review support for clinical trial plans ▲ a waiver of new drug application fees.
If final marketing approval is obtained, the company can also secure, in principle, seven years of market exclusivity in the United States for the same drug and indication.
AML, the target of this designation, is a blood cancer in which white blood cells in the bone marrow turn into cancer cells and proliferate rapidly. Early symptoms are not distinct, making early diagnosis difficult, and the disease tends to worsen quickly.
Traditionally, highly toxic anticancer drugs or bone marrow transplants have been used, but side effects and the risk of relapse have been cited as limitations. Targeted therapies have emerged in recent years, but drug resistance can develop during treatment.
SillaJen expects that, because BAL0891 simultaneously targets key factors involved in cancer cell division, it could offer a new treatment option for AML patients who have developed resistance to existing therapies.
A SillaJen official said, "Because it was designated as an orphan drug from the early clinical stage, we see this as a degree of recognition of BAL0891's medical importance and potential," adding, "based on this achievement, we will do our best to deliver more positive results in the remaining clinical schedule."