FAB Supports Market Launch of the Class 1 Innovative Drug Gecacitinib Hydrochloride Tablets, Self-Developed by ZELGEN

On May 29, 2025, the latest announcement from the National Medical Products Administration (NMPA) revealed that ZELGEN Biopharmaceuticals' self-developed Gecacitinib Hydrochloride Tablets have been approved for marketing. The approved indication is "for the treatment of adult patients with intermediate or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), and post-essential thrombocythemia myelofibrosis (PET-MF), to address disease-related splenomegaly or disease-related symptoms."

FAB, as China's leading medical imaging CRO, provided comprehensive Independent Review Committee (IRC) services for the Phase III clinical study of this drug.

Source: NMPA

Myelofibrosis (MF) is a diffuse myelofibrotic disorder characterized by the replacement of normal blood cell precursor cells in the bone marrow with fibrous tissue, leading to poikilocytosis, anemia, and splenomegaly. MF patients can be stratified into low-risk, intermediate-1-risk, intermediate-2-risk, and high-risk groups based on the International Prognostic Scoring System (IPSS) and the Dynamic International Prognostic Scoring System (DIPSS). According to DIPSS, median survival for intermediate-2-risk and high-risk MF patients is approximately 4 years and 1.5 years, respectively, severely impacting patients' quality of life and lifespan.

Gecacitinib is a novel JAK inhibitor, classified as a Class 1 innovative drug, for which ZELGEN holds independent intellectual property rights. As a JAK inhibitor, gecacitinib's molecular mechanism of action for treating MF involves inhibiting the activity of non-receptor tyrosine Janus-associated kinases JAK1, JAK2, JAK3, and TYK2, thereby blocking the JAK-STAT signaling pathway to reduce inflammation and splenomegaly. Additionally, gecacitinib also inhibits activin receptor type 1 (ACVR1); by suppressing ACVR1 activity, it can improve iron metabolism imbalance and anemia.

This approval was primarily based on "A Randomized, Double-blind, Double-dummy, Parallel-controlled, Multicenter Phase III Clinical Trial Evaluating the Efficacy and Safety of Gecacitinib Hydrochloride Tablets Versus Hydroxyurea Tablets in Intermediate/High-Risk Myelofibrosis Patients". The primary efficacy endpoint results showed that 72.3% of subjects treated with Gecacitinib Hydrochloride achieved ≥35% reduction in spleen volume (SVR35) from baseline at week 24. Other endpoints, including clinical improvement, anemia response, hemoglobin improvement, and disease symptom scores, also demonstrated significant benefits. Furthermore, results from the "Phase IIB Clinical Trial on the Safety and Efficacy of Gecacitinib Hydrochloride Tablets in Ruxolitinib-Intolerant Myelofibrosis Patients" showed a SVR35 rate of 43.2% at week 24, and the "Phase II Clinical Trial on the Efficacy of Gecacitinib Hydrochloride Tablets in Ruxolitinib-Refractory or Relapsed Myelofibrosis Patients" reported a SVR35 rate of 32.4% at week 24. Clinical studies also indicated that Gecacitinib Hydrochloride has a favorable safety and tolerability profile in these patient populations.

Source: ZELGEN

For many years, FAB and ZELGEN have maintained a long-term, stable collaborative relationship, with FAB continuously serving as the Independent Review Committee (IRC) for ZELGEN's critical projects.

This study enrolled a total of 105 MF patients. Given the complexity and specificity of this indication, the project was spearheaded and overseen throughout by Dr. Luxia Liang, CEO of FAB. Leveraging extensive industry experience, a senior expert team, and a highly efficient project team, FAB successfully completed the core task of central image review with high quality. This included rigorous quality assessment of baseline images for all subjects and systematic execution of follow-up efficacy evaluations throughout the study period, ensuring the objectivity and reliability of efficacy data.

During project execution, FAB prioritized image data quality control as the project cornerstone. The team actively participated in designing the image acquisition protocol and trained relevant personnel at investigator meetings, emphasizing the unique aspects of this project. By scientifically developing independent review charters, customizing the review system, proactively planning and efficiently coordinating reviewer schedules, FAB ultimately ensured outstanding overall quality in image review completion. This provided solid support for the reliability of the clinical trial endpoints.

At the critical juncture of the NMPA inspection, when faced with challenging issues, the FAB team demonstrated a high degree of professionalism and a spirit of proactive collaboration. They fully supported the sponsor in successfully addressing the inspectors' inquiries and providing explanations. The team's rapid response capability and dedicated work attitude earned high praise from the sponsor.

The approval of this drug marks a significant breakthrough in China's new drug R&D – as the first domestically developed innovative JAK inhibitor approved for MF treatment. Its success also fully demonstrates FAB's top-tier capabilities and exceptional project execution in managing imaging assessments for specialized indications, marking another important milestone in the company's history. Notably, this achievement brings the total number of approvals supported by FAB to surpass 40, setting a new record high!

FAB extends its warmest congratulations to ZELGEN on this major achievement! We look forward to continuing our partnership on the path of future pharmaceutical innovation, achieving further successes, and bringing benefits to patients worldwide.