ABL Bio said on the 8th that "Torbeximig," a cholangiocarcinoma treatment under development by its global partner Compass Therapeutics, received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA).
The orphan drug designation system is intended to encourage the development of treatments for diseases affecting fewer than 200,000 patients. Upon designation, up to seven years of market exclusivity is granted in the United States, along with benefits such as a tax credit, partial waiver of review fees, clinical trial subsidies, and development support from regulatory agencies.
Torbeximig is a bispecific antibody therapy developed by ABL Bio and licensed to Compass in the United States. It simultaneously blocks the signaling pathways (DLL4 and VEGF-A) involved in tumor angiogenesis, thereby inhibiting tumor blood vessel formation. Preclinical and clinical studies have shown that dual inhibition of the two pathways suppresses tumor growth.
Compass is conducting phase 2/3 trials combining the chemotherapy drug "paclitaxel" for second-line cholangiocarcinoma patients. A data release on key endpoints, including overall survival (OS) and progression-free survival (PFS), is scheduled this month. OS indicates how much longer patients actually lived, and PFS refers to the period during which the disease does not worsen.
Earlier, the candidate received priority review (fast track) designation from the FDA in Apr. 2024. The company expects the new orphan drug designation to have a positive impact on the approval process.
ABL Bio is developing multiple pipelines based on its bispecific antibody platform "Grabody." Key candidates are in clinical trials in the United States, China, Australia, and Korea. The company plans to present additional clinical data at global conferences in the second half of this year.