NDAORALCAPSULEPriority Review
Approved
Dec 2023
Lifecycle
Growth
Competitive Pressure
0/100
Clinical Trials
21
Data completeness
4/8 — Partial
Priority Review
Drug data last refreshed 3d ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
FABHALTA (iptacopan) is an oral small-molecule complement Factor B inhibitor approved in December 2023 for rare hemolytic disorders. It treats Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) by blocking proximal complement cascade activation. This represents a novel oral alternative to existing intravenous complement inhibitor therapies.
Growth15.2 years to LOE
Early-stage growth product with 15+ years patent protection; expect expanding commercial teams and market development opportunities.
Mechanism of Action
Mechanism of action data is being enriched from DailyMed and FDA sources. Check back soon for updated drug intelligence.
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (21)
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
Started Jul 2026
21 enrolled
Paroxysmal Nocturnal HemoglobinuriaC3 Glomerulopathy
IgA Nephropathy Insights From Treatment Experience Among Patients Receiving Iptacopan and/or Atrasentan Using Primary Data Collection
Started May 2026
80 enrolled
IgA Nephropathy
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Started Jan 2026
50 enrolled
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy
Started Jan 2026
30 enrolled
Thrombotic MicroangiopathyHematopoietic Stem Cell Transplantation (HSCT)
Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN
Started Nov 2025
31 enrolled
Primary Immunoglobulin A Nephropathy (IgAN)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.