Accro Bioscience's AC-101 Included in NMPA CDE Caring Plan-Extension for Blau Syndrome

Accro Bioscience, a clinical-stage biotechnology company, has announced that its drug AC-101 tablets have been included in the National Medical Products Administration (NMPA) Center for Drug Evaluation’s (CDE) "Caring Plan-Extension" pilot program for rare disease drug development. The inclusion is for the treatment of Blau syndrome, a rare hereditary autoinflammatory disease.

The CDE's pilot program aims to expedite the development and approval of drugs for rare diseases, offering a streamlined pathway for drug candidates. AC-101, a novel, selective RIPK2 inhibitor, is being developed for the treatment of moderate-to-severe ulcerative colitis (UC). The drug has already demonstrated favorable safety and pharmacokinetic/pharmacodynamic (PK/PD) profiles in Phase I studies conducted in Australia and China.

AC-101's development for Blau syndrome is significant as the disease is caused by pathogenic variants in the NOD2 gene, leading to a triad of symptoms including granulomatous dermatitis, arthritis, and uveitis. The disease typically manifests in early childhood and can lead to joint deformity and functional impairment, as well as severe visual impairment if left untreated.

In June 2026, the U.S. Food and Drug Administration (FDA) had granted AC-101 Rare Pediatric Disease (RPD) designation for the treatment of Blau syndrome. This designation provides AC-101 with a priority review voucher, which can be used to expedite the FDA's review process for a subsequent drug application.

Accro Bioscience's AC-101 has completed a Phase Ib/IIa proof-of-concept study in Chinese patients with moderate-to-severe UC. The results from this study will be presented at UEG Week 2026, a major conference in the field of gastroenterology. The company's pipeline includes first- and best-in-class compounds targeting regulatory cell death and inflammation, with the potential to bring innovative therapies to patients suffering from inflammatory and autoimmune diseases.

The inclusion of AC-101 in the CDE's pilot program is a significant step in the drug's development for Blau syndrome. The program is designed to provide a faster and more efficient pathway for the approval of drugs for rare diseases, which often lack adequate treatment options. This move by the NMPA is part of a broader effort to improve the drug development process for rare diseases in China, ensuring that patients have access to innovative treatments more quickly.

Accro Bioscience's focus on developing novel therapeutics for inflammatory and autoimmune diseases through the targeting of molecular mechanisms of regulatory cell death positions the company as a leader in this field. The company's robust pipeline of first- and best-in-class compounds offers the potential to bring transformative therapies to patients, addressing unmet medical needs in a variety of disease areas.