By Fineline Cube 
Swiss pharmaceutical giant Novartis (NYSE: NVS) announced that its first‑in‑class oral inhibitor Iptacopan (Fabhalta) has received additional approval from the China National Medical Products Administration (NMPA) for the treatment of IgA nephropathy (IgAN).
What is Iptacopan?
- Target – Selectively inhibits complement factor B, a key driver of the alternative complement pathway.
- Current Indications –
- Paroxysmal nocturnal hemoglobinuria (PNH) – Approved April 2024; expanded May 2025 to all adult PNH patients, including those previously treated with complement inhibitors.
- C3 glomerulopathy (C3G) – Approved April 2025; reduces proteinuria in adult patients.
- New Indication – IgA nephropathy (IgAN) – First oral complement‑inhibitor therapy approved for this common glomerular disease.
Clinical Evidence Supporting the IgAN Approval
| Parameter | Iptacopan | Placebo | % Reduction |
|---|---|---|---|
| Morning urine protein‑to‑creatinine ratio (UPCR) at 2 weeks | Rapid decline | No change | – (rapid efficacy) |
| Proteinuria at 9 months | 38.3% ↓ | 0% ↓ | 38.3 % |
| Safety/Tolerability | Mild adverse events; no serious safety signals | – | – |
The pivotal Phase 3 study demonstrated a 38.3 % reduction in proteinuria after 9 months, with a favorable safety profile and rapid onset of action—key factors that enabled the NMPA’s approval.
Broader Development Pipeline
Novartis is advancing Iptacopan across a spectrum of complement‑mediated disorders, including:
- Atypical hemolytic uremic syndrome (aHUS)
- Immune‑complex membranoproliferative glomerulonephritis (IC‑MPGN)
- Lupus nephritis (LN)
This portfolio underscores Novartis’s commitment to transforming complement‑driven diseases worldwide.
Market Implications
- First Oral Therapy – Iptacopan’s oral route offers a convenient alternative to injectable complement inhibitors, potentially improving patient adherence.
- China’s Growing Biopharma Market – The IgAN approval positions Novartis to capture a large patient pool in China, where IgAN prevalence is rising.
- Competitive Edge – By expanding into multiple renal indications, Novartis strengthens its foothold against other complement‑targeted agents.
Next Steps
- Launch in China – Commercial rollout slated for Q4 2025.
- Real‑World Evidence – Post‑marketing studies to confirm long‑term efficacy and safety.
- Global Expansion – Leverage China data to support regulatory submissions in the EU and US for IgAN and other renal indications.-Fineline Info & Tech