Iptacopan
Paroxysmal nocturnal hemoglobinuria (PNH), IgA nephropathy and C3 glomerulopathy
- CAS No.
- 1644670-37-0
- Indication Class
- Cardiovascular / Renal
- Patent Expiry
- To be assessed
- Therapeutic Area
- Rare Disease / Nephrology
- Target / Mechanism
- CFB
- Labeled Purity
- 98%+
- Registration Readiness
- GMP/DMF
- Commercialization Tier
- API (Commercialized)
- Linked Intermediates
- Confirm per inquiry
Key Intermediates for This API
Order: listed intermediates that accept enquiries first; fragments shared across several APIs rank higherNo intermediate entries are on file for this API yet. You can still enquire by CAS number — we will route it to the supply side for manual sourcing.
Listing Logic · Why It's Worth Listing
The first oral complement-factor-B inhibitor, converting PNH from infusion to oral therapy and expanding into nephrology, a high-value rare-disease molecule.
Rare Disease / Nephrology · Marketed; available for immediate sourcing
GMP/DMF-ready; supports regulatory filing and audit in target markets
Intermediates confirmed per inquiry
Product Description
Iptacopan represents a significant breakthrough in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Approved in late 2023, it is the first oral, targeted factor B inhibitor for this rare and life-threatening blood disease.
Supply source: Unibest · Iptacopan, sourced, vetted and delivered by Unibest. Therapeutic area, indication and listing rationale are qualitative summaries based on public drug knowledge for business reference; actual registration availability and quality specifications are subject to DMF/GMP documentation and the technical agreement confirmed at order.