Wuhan, China — Wuhan Createrna Science and Technology Development Co., Ltd. (“Createrna”) announced that MY008211A Tablets, a Class 1 innovative chemical drug independently developed by the company, has been officially included in the Priority Review List by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) following the completion of the public comment period. The drug is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy.

Concurrently, the New Drug Application (NDA) for MY008211A Tablets was formally accepted by the CDE on October 17, 2025, entering the regulatory review phase.

First-in-Class Domestically Developed CFB Inhibitor

MY008211A is an oral complement factor B (CFB) inhibitor independently developed by Createrna. It represents the company’s first innovative drug to enter the NDA review stage and is the first domestically developed CFB inhibitor in China.

Phase II Clinical Data

In December 2024, Createrna presented results from a multicenter, open-label, dose-finding Phase II clinical study (NCT06050226) evaluating MY008211A Tablets as monotherapy in complement inhibitor-naïve PNH patients at the American Society of Hematology (ASH) Annual Meeting. Key findings included:

• At Day 84, all 34 subjects (100%) across all dose cohorts achieved the primary endpoint of hemoglobin increase ≥20 g/L from baseline, without transfusion support.

• All patients remained transfusion-free throughout MY008211A treatment.

In December 2024, MY008211A Tablets also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of PNH.

About Paroxysmal Nocturnal Hemoglobinuria

PNH is an acquired hemolytic disease—a chronic, progressive, life-threatening, rare multisystem disorder. It was included in China’s First National Rare Disease Catalogue in 2018. Currently, no domestically developed CFB-targeted inhibitor has been approved for marketing in China.

Priority Review Pathway

Inclusion in the Priority Review program means the NDA for MY008211A will enter an expedited evaluation track. Under this program, the CDE will allocate review resources on a priority basis, and the National Drug Inspection Center, drug testing institutions, and the Chinese Pharmacopoeia Commission will prioritize on-site inspections, registration testing, and generic name approval. The statutory review timeline will be shortened from the standard 200 working days to 130 working days, accelerating the path to market approval and benefiting PNH patients sooner.