Jiangsu Hengrui Pharmaceuticals Co., Ltd. (SHA: 600276, HKG: 1276) announced that China’s National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for SHR-1918 injection, a Category 1 novel drug, and granted priority review status. The indication covers adult and adolescent patients aged 12 and above with homozygous familial hypercholesterolemia (HoFH), offering a potential new treatment option for this rare and severe genetic lipid disorder.
Target:ANGPTL3 (angiopoietin‑like protein 3) – liver‑secreted protein regulating lipid metabolism
Mechanism: Inhibition of ANGPTL3 activity → reduction of serum triglycerides (TG) and LDL‑cholesterol (LDL‑C)
Development Status: NDA under priority review; Phase III completed
Clinical Profile: Significant efficacy with favorable safety demonstrated in clinical studies
Clinical Evidence Summary
Endpoint
SHR-1918 Performance
LDL‑C Reduction
Significant lowering demonstrated
Triglyceride Reduction
Significant TG lowering
Safety Profile
Favorable tolerability
Patient Population
HoFH patients with severe, refractory hypercholesterolemia
Strategic Implications
Rare Disease Focus: HoFH is an ultra‑rare genetic disorder (prevalence ~ 1 in 300,000) with limited treatment options; SHR-1918 addresses critical unmet need for patients failing statins and PCSK9 inhibitors.
ANGPTL3 Validation: Targeting ANGPTL3 (vs. PCSK9 or LDL receptor) offers a complementary mechanism for lipid lowering, with potential in broader dyslipidemia populations pending future studies.
Priority Review Acceleration: NMPA priority review status reflects HoFH severity and SHR-1918’s Breakthrough Therapy pedigree, potentially shortening time‑to‑approval.
Hengrui Innovation Pipeline: As a Category 1 novel biologic, SHR-1918 demonstrates Hengrui’s internal R&D capabilities beyond its established generics and oncology franchise.
Market Context
Factor
Impact
HoFH Market Size
Ultra‑rare but high‑value indication; annual treatment costs typically $300K+ per patient
Competitive Landscape
Regeneron’s Evkeeza (evinacumab) is approved ANGPTL3 antibody; SHR-1918 would be first‑in‑class in China
China Rare Disease Policy
NMPA priority review and Breakthrough Designation reflect supportive regulatory environment for orphan drugs
Pipeline Expansion
Successful HoFH approval could support broader dyslipidemia indications (severe hypertriglyceridemia, HeFH)
Forward‑Looking Statements This brief contains forward‑looking statements regarding NMPA approval timelines, commercial launch expectations, and label expansion potential for SHR-1918. Actual results may differ due to risks including regulatory review outcomes, pricing negotiations, and competitive dynamics in the lipid‑lowering market.-Fineline Info & Tech
By Fineline Cube