FABHALTA Set to Disrupt Complement Inhibitor Market with Oral Convenience and Strong Efficacy | DelveInsight

With future approvals of FABHALTA, the market size is expected to expand significantly in the coming years, driven by its potential to address unmet medical needs across multiple conditions like IgAN, PNH, and other complement-driven diseases.

LAS VEGAS, April 29, 2025 /PRNewswire/ -- DelveInsight's "FABHALTA Market Size, Forecast, and Market Insight Report" highlights the details around FABHALTA, an oral complement factor B inhibitor used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), primary immunoglobulin A nephropathy (IgAN), and C3 glomerulopathy (C3G). The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of FABHALTA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Novartis' FABHALTA (iptacopan) Overview

Iptacopan is an innovative, first-in-class oral small molecule that reversibly inhibits factor B—a crucial serine protease in the alternative pathway of the complement system. By targeting this point upstream of the C5-terminal pathway, iptacopan effectively prevents both intravascular and extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). Its selective mechanism allows it to modulate alternative pathway dysfunction without compromising the body's defense against infections via other complement pathways, thus lowering the risk of infection.

FABHALTA is the brand name of iptacopan and is prescribed to:

• Treat adults with PNH.
• Reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of rapid disease progression.
• Treatment of adults with C3 glomerulopathy (C3G) to reduce proteinuria.

Iptacopan is also being investigated for other complement-mediated diseases, including myasthenia gravis and atypical hemolytic uremic syndrome (aHUS)—all representing areas of high unmet medical need. It may become the first complement pathway inhibitor capable of slowing disease progression in these conditions. Reflecting its promise, iptacopan has received orphan drug designations from both the FDA and EMA for C3G and PNH, along with EMA PRIME status for C3G and EMA orphan designation for IgAN, supported by encouraging Phase II clinical data and disease prevalence.

Learn more about FABHALTA projected market size for PNH and IgAN @ FABHALTA Market Potential 

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, non-cancerous blood disorder caused by a clonal defect, leading to the absence of GPI-anchored complement regulatory proteins on the surface of blood-forming cells. This deficiency makes the cells vulnerable to destruction by the complement system. In 2024, approximately 16,000 diagnosed prevalent cases of PNH were reported across the 7MM, with the United States accounting for the largest share. The number of cases is expected to grow at a moderate CAGR through 2034.

Treatment for PNH primarily focuses on complement inhibition, with FDA-approved therapies such as SOLIRIS, ULTOMIRIS, and EMPAVELI serving as the standard of care. However, concerns remain around the safety profiles of eculizumab and ravulizumab. Recently approved agents targeting factor B and D—such as FABHALTA (iptacopan), VOYDEYA (danicopan), and PIASKY (crovalimab)—seek to provide more effective and safer treatment options. 

The market size of PNH in the 7MM was more than USD 1.2 billion in 2024. The United States accounted for the largest market share with approximately 80% in 2024 among 7MM. Overall, the PNH treatment landscape is expected to evolve significantly between 2025 and 2034, driven by the introduction of innovative therapies currently in clinical development.

Discover more about the PNH market in detail @ Paroxysmal Nocturnal Hemoglobinuria Market Report

IgA Nephropathy (IgAN) is an autoimmune condition that targets the kidneys, disrupting blood filtration within the small renal blood vessels. This disease is triggered by an abnormal protein that harms the glomeruli—the kidneys' filtering units. DelveInsight estimates that there were approximately prevalent cases of IgAN across the 7MM in 2024. These diagnosed prevalent cases are projected to rise by 2034 at a CAGR of 0.6%. Traditional treatment approaches primarily involve ACE inhibitors and angiotensin II receptor blockers (ARBs), which help control symptoms such as high blood pressure.

Currently approved therapies for IgAN include TARPEYO/KINPEYGO (budesonide), FABHALTA (iptacopan), FILSPARI (sparsentan), VANRAFIA (atrasentan). The treatment landscape is expected to undergo substantial transformation between 2024 and 2034, driven by the introduction of innovative therapies. According to DelveInsight's analysis, the IgAN market in the 7MM was valued at approximately USD 730 million in 2024. Over the forecast period from 2025 to 2034, this market is projected to grow at a CAGR of 30.5%.

Given that targeted treatments for IgA nephropathy were virtually nonexistent until recently—and considering the critical need for options that delay progression to end-stage kidney disease (ESKD)—any major therapeutic advancement is likely to significantly reshape the market during the forecast period.

For a comprehensive view of the IgAN market, check out the IgA Nephropathy Market Assessment

Emerging Competitors of FABHALTA

Emerging PNH therapies include Zaltenibart (OMS906) (Omeros), Pozelimab (Regeneron Pharmaceuticals), Ruxoprubart (NM8074) (NovelMed), and others, reflecting a dynamic evolution in treatment strategies. In February 2024, NovelMed announced that the US FDA had granted ODD to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of PNH.

The IgA nephropathy pipeline possesses some drugs in early, mid, and late-stage developments to be approved in the near future. The emerging landscape holds a diverse range of therapeutic alternatives for treatment, including APRIL inhibitor (Sibeprenlimab, Zigakibart), Complement inhibitor (IONIS-FB-LRx/RG6299), BAFF and APRIL antagonist (Povetacicept), C5 complement inhibitor (ULTOMIRIS), BLyS inhibitor and APRIL inhibitor (Atacicept), Anti CD38 (Felzartamab, Mezagitamab), Complement factor D inhibitor (Vemircopan), Targets complement protein Bb (Ruxoprubart), Binding to and blocking both C3d and factor H (ADX-097), Blocks both the alternative (Factor H) and terminal (C5) complement pathways (KP104), Soluble urokinase plasminogen activator receptor (suPAR) inhibitors (WAL0921), C3 inhibitor (ARO-C3), and others in different lines of treatment. The expected launch of these therapies shall further create a positive impact on the market.

The emerging pipeline of C3G holds a few significant products in development by prominent key players such as Apellis Pharmaceuticals and Arrowhead Pharmaceuticals, coming up with interventions such as pegcetacoplan (APL-2) and ARO-C3, respectively.

To know more about the number of competing drugs in development, visit @ FABHALTA Market Positioning Compared to Other Drugs

Key Milestones of FABHALTA

• In April 2025, Novartis Canada was proud to share that FABHALTA is now accessible nationwide for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) experiencing hemolytic anemia. As the first and only oral monotherapy approved for PNH, FABHALTA received authorization from Health Canada in January 2025.
• In March 2025, Novartis announced that the FDA had approved the oral drug FABHALTA (iptacopan) for treating adults with C3 glomerulopathy (C3G) to help reduce proteinuria. This marks the first and only approved treatment available for this condition.
• In August 2024, Novartis received FDA accelerated approval for FABHALTA (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN).
• In December 2023, Novartis received FDA approval for FABHALTA (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH).
• In December 2023, Novartis' investigational iptacopan Phase III study demonstrated clinically meaningful and statistically significant proteinuria reduction in patients with C3 glomerulopathy (C3G).

FABHALTA Patent Details

FABHALTA is a drug owned by Novartis Pharmaceuticals. It is protected by 3 US drug patents filed from 2023 to 2024, out of which none have expired yet. FABHALTA's patents will be open to challenges from 06 December 2027. Based on its patents and exclusivities, its generic launch date is estimated to be Jul 15, 2041.

Discover how FABHALTA is shaping the IgAN treatment landscape @ FABHALTA IgAN

FABHALTA Market Dynamics

FABHALTA is a first-in-class oral factor B inhibitor developed by Novartis for the treatment of PNH. Its approval marks a significant shift in the competitive landscape of the PNH market, which has historically been dominated by intravenous complement inhibitors such as SOLIRIS and ULTOMIRIS from AstraZeneca's Alexion unit. FABHALTA's oral route of administration, coupled with its ability to control both intravascular and extravascular hemolysis, offers a differentiated clinical profile that addresses some of the key limitations of current therapies, potentially expanding the PNH market and redefining the standard of care.

The market uptake of FABHALTA is expected to be driven by several factors: patient and physician preference for oral therapies, improved quality of life due to reduced transfusion dependence, and its favorable efficacy and safety profile. As an oral agent, FABHALTA reduces the burden associated with biweekly or monthly infusions, a critical consideration for patient adherence and long-term disease management. Moreover, its ability to target the alternative complement pathway upstream of C5 provides a more holistic control of hemolysis, including extravascular components often insufficiently addressed by current C5 inhibitors.

However, FABHALTA's success will be influenced by various market dynamics, including pricing strategies, payer acceptance, and competition from emerging therapies. While Novartis is leveraging its strong global footprint to ensure rapid access, the high cost of novel therapies could pose reimbursement challenges in certain geographies. Additionally, other pipeline agents targeting upstream components of the complement cascade (e.g., factor D or C3 inhibitors) or offering similar oral convenience may intensify competition, requiring continuous differentiation through real-world evidence and long-term outcomes.

Strategic partnerships, post-marketing studies, and label expansions—such as use in other complement-mediated diseases (e.g., C3G, aHUS, IgAN)—could further enhance FABHALTA's commercial potential and market footprint. As the complement therapeutics space grows increasingly crowded and sophisticated, Novartis will need to maintain a robust lifecycle management strategy to preserve its competitive edge and maximize market penetration.

Dive deeper to get more insight into FABHALTA's strengths & weaknesses relative to competitors @ FABHALTA Market Drug Report

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SOURCE DelveInsight Business Research, LLP