Accro Bioscience's AC-101 Tablets Enter NMPA CDE "Caring Plan-Extension" Pilot for Blau Syndrome
核心洞察
China's NMPA Center for Drug Evaluation has included Accro Bioscience (搜索)'s AC-101 tablets in its Rare Diseases Innovative Drug Development Encouragement Pilot Program, with Blau syndrome (搜索) as the proposed indication.
The inclusion follows the June 2026 FDA Rare Pediatric Disease designation for AC-101 in Blau syndrome (搜索), a rare hereditary autoinflammatory disease driven by pathogenic NOD2 gene variants.
AC-101 is a novel selective RIPK2 (搜索) inhibitor from Accro's regulatory cell death platform, and is also in development for moderate-to-severe ulcerative colitis (搜索).
Accro Bioscience (搜索) announced that its investigational AC-101 tablets have been included in the Rare Diseases Innovative Drug Development Encouragement Pilot Program — the "Caring Plan-Extension" (known in Chinese as "关爱计划-延伸") — run by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA). The proposed indication for the inclusion is Blau syndrome (搜索). The announcement, dated September 13, 2026, states that the program's relevant public notice period ended on September 11, 2026, with the inclusion taking effect on September 14, 2026 China Standard Time.
The Chinese regulatory milestone follows a U.S. action earlier in 2026: in June 2026, the U.S. Food and Drug Administration granted AC-101 Rare Pediatric Disease (RPD) designation for the treatment of Blau syndrome (搜索) (BS).
A Selective RIPK2 Inhibitor From a Regulatory Cell Death Platform
AC-101 is described as a novel, selective RIPK2 (搜索) inhibitor derived from Accro Bioscience (搜索)'s proprietary drug discovery platform targeting regulatory cell death and inflammation. Receptor interacting protein kinase 2 (RIPK2) is a key mediator in the NOD signaling pathway, and dysregulation of NOD/RIPK2 signaling is associated with multiple inflammatory and autoimmune diseases, including inflammatory bowel disease (搜索) (IBD).
Beyond Blau syndrome (搜索), the company is developing AC-101 for moderate-to-severe ulcerative colitis (搜索) (UC). According to the announcement, AC-101 has completed Phase I studies in healthy volunteers in Australia and China, demonstrating a favorable safety and PK/PD profile. A Phase Ib/IIa proof-of-concept study in Chinese patients with moderate-to-severe UC has also been completed, and the efficacy and safety results from that study will be presented at UEG Week 2026.
Blau Syndrome: A Rare NOD2-Driven Autoinflammatory Disease
Blau syndrome (搜索) is a rare hereditary autoinflammatory disease caused by pathogenic variants in the NOD2 gene. It usually begins in early childhood and can involve multiple systems, including the skin, joints and eyes. Its classic clinical presentation is the triad of granulomatous dermatitis, arthritis and uveitis.
The disease burden described by the company centers on two organ systems. Joint involvement may lead to joint deformity and functional impairment over time, while ocular involvement can cause uveitis that, if not controlled promptly, may lead to severe visual impairment or even blindness.
Company Profile
Accro Bioscience (搜索) Inc. is a clinical-stage biotechnology company pioneering the discovery, development and commercialization of novel therapeutics for inflammatory and autoimmune diseases by targeting molecular mechanisms of regulatory cell death. The company states that its pipeline of first- and best-in-class compounds offers the potential to bring innovative, paradigm-shifting therapies to patients.