Jiangsu Hengrui Secures Priority Review for HRS‑5965 in China’s Rare‑Disease Portfolio

Jiangsu Hengrui Pharmaceuticals (SHA: 600276, HKG: 1276) announced that the National Medical Products Administration (NMPA) has granted Priority Review status to its investigational HRS‑5965 capsule, targeting adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not yet received complement‑inhibitor therapy.
The decision positions HRS‑5965 as a potential first‑in‑class complement factor B inhibitor in the rapidly evolving PNH market.

Therapeutic Profile

Mechanism of Action

• HRS‑5965 is a selective complement factor B inhibitor that dampens the assembly of the membrane‑attack complex (MAC), thereby reducing both intravascular and extravascular hemolysis.
• In pre‑clinical and early‑phase trials, the molecule has shown the capacity to elevate hemoglobin concentrations and alleviate anemia in PNH patients who lack prior exposure to complement inhibitors.

Competitive Landscape

• The only therapy worldwide with a similar target is Novartis’ iptacopan hydrochloride capsules (Fabhalta), approved outside of China.
• No complement‑factor B inhibitor has yet received approval for PNH in China, giving HRS‑5965 a distinctive competitive edge pending approval.

Paroxysmal Nocturnal Hemoglobinuria: A Rare‑Disease Catalyst

• PNH is an acquired, lifelong hemolytic disorder driven by uncontrolled complement activation due to deficiencies in CD55 (Decay Accelerating Factor) and CD59 (Membrane Inhibitor of Reactive Lysis).
• Clinical hallmarks include intravascular hemolysis, progressive hematopoietic failure, and a heightened propensity for thrombosis.
• Because of its low prevalence, PNH appears on China’s First National List of Rare Diseases, ensuring eligibility for expedited regulatory pathways and special reimbursement schemes.

Implications for Jiangsu Hengrui

• Revenue Potential – The PNH market in China is modest (estimated 5,000–6,000 patients nationwide), yet the disease’s inclusion on the national rare‑disease list unlocks special pricing and accelerated market access once approval is granted.
• Regulatory Momentum – Priority Review typically shortens the post‑approval review period by 6–12 months; Hengrui is now poised to move from a fast‑track investigational product to an approved therapy much sooner than the standard 12‑month timeline.
• Strategic Positioning – With the single existing complement‑inhibitor (iptacopan) approved only abroad, HRS‑5965 could capture a sizeable share of the domestic rare‑disease market and serve as a launchpad for future complement‑inhibitor indications (e.g., atypical hemolytic uremic syndrome, myasthenia gravis).

Conclusion

Jiangsu Hengrui’s receipt of Priority Review for HRS‑5965 underscores the NMPA’s commitment to fast‑tracking therapies for rare diseases while simultaneously advancing the company’s portfolio against a backdrop of complement‑inhibitor expansion. The next fiscal quarter will be pivotal as bridging studies in China move to confirm efficacy and safety in the target PNH cohort.-Fineline Info & Tech