By Fineline Cube 
Shanghai CirCode Biomed Co. Ltd. today announced that its proprietary circular‑RNA (circRNA) therapeutic HM2003 Injection has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Thromboangiitis Obliterans (TAO), also known as Buerger’s disease.
What Is Thromboangiitis Obliterans?
- Chronic, non‑atherosclerotic vasculitis that targets medium and small arteries and veins of the limbs.
- Characterized by intense inflammatory cell infiltration, thrombosis, and progressive occlusion of the vascular lumen.
- Leads to Chronic Limb‑Threatening Ischemia (CLTI), especially in young (< 50 yrs) smokers.
- No disease‑specific therapies currently address the underlying inflammatory and thrombotic pathology, creating a critical unmet medical need.
HM2003 Injection – A Novel circRNA Approach
| Feature | Detail |
|---|---|
| Drug class | Circular RNA (circRNA) therapeutic |
| Mechanism | Encodes a synthetic peptide that modulates endothelial repair and reduces inflammation; delivered via sub‑cutaneous injection. |
| Clinical promise | Early‑phase data suggest rapid improvement in limb perfusion and reduction in inflammatory biomarkers. |
| Orphan status | FDA ODD confers 7‑year market exclusivity, tax credits, and potential priority review for subsequent approvals. |
Strategic Significance of FDA Orphan Designation
- Accelerated development pathway: Streamlined regulatory review and potential fast‑track filing for phase II/III studies.
- Commercial advantages: Market exclusivity and incentives lower the cost of bringing a therapy to underserved patient populations.
- Global impact: Positions Shanghai CirCode as a pioneer in circRNA therapeutics for vascular diseases, opening doors to collaborations with U.S. academic and industry partners.
Next Steps
- Phase II clinical trial: Planned enrollment of 120 TAO patients in the U.S. and China to evaluate efficacy and safety.
- Regulatory strategy: Submit a Biologics License Application (BLA) in 2027, leveraging the ODD status for priority review.
- Commercial planning: Engage with payers and patient advocacy groups to ensure rapid access upon approval.
Forward‑Looking Statements
This release contains forward‑looking statements that involve risks and uncertainties. Actual results may differ materially.-Fineline Info & Tech