Suzhou – – September 16, 2026 -- Accro Bioscience said AC-101 tablets have been included in the National Medical Products Administration's Center for Drug Evaluation (CDE) "Caring Plan-Extension" pilot program, targeting Blau syndrome as the proposed indication, with the public notice period closing September 11, 2026.
NMPA pilot program aims to accelerate rare disease drug development in China
The Caring Plan-Extension pilot, run by China's CDE, is designed to encourage innovative drug development for rare diseases. Accro Bioscience confirmed the inclusion took effect September 14, 2026, China Standard Time (UTC+8).
FDA granted AC-101 Rare Pediatric Disease designation three months earlier
The U.S. Food and Drug Administration awarded AC-101 Rare Pediatric Disease (RPD) designation for Blau syndrome in June 2026, ahead of the Chinese regulatory step.
AC-101 is a selective RIPK2 inhibitor with a separate ulcerative colitis program
AC-101 targets receptor interacting protein kinase 2 (RIPK2), a key mediator of the NOD signaling pathway implicated in inflammatory bowel disease and other autoimmune conditions. The compound has completed Phase I studies in healthy volunteers in Australia and China, and a Phase Ib/IIa proof-of-concept trial in Chinese patients with moderate-to-severe ulcerative colitis has also concluded, with efficacy and safety data scheduled for presentation at UEG Week 2026.
Blau syndrome drives progressive joint deformity and vision loss in children
Blau syndrome is a rare hereditary autoinflammatory disorder caused by pathogenic variants in the NOD2 gene, typically presenting in early childhood with a triad of granulomatous dermatitis, arthritis and uveitis. Untreated joint involvement can progress to deformity and functional impairment, while uncontrolled uveitis risks severe visual impairment or blindness.