Hope on the Horizon: Nephrologists Emphasize Need for Innovative C3G Treatments as Novartis and Apellis Prepare Therapies for FDA Submission

EXTON, PA, Nov. 15, 2024 (GLOBE NEWSWIRE) — Complement 3 glomerulopathy (C3G) is a rare, progressive kidney disease characterized by inflammation and kidney damage. Patients with C3G often experience high blood pressure, protein or blood in the urine, swelling, fatigue, and recurrent infections. While existing treatments such as steroids and immunosuppressants offer short-term relief, they come with risks and side effects. As patients have limited long-term solutions, physicians identify an urgent need for innovative therapies. 

Spherix has been tracking the C3G market for over four years in both the U.S. and Europe. The studies have provided meaningful insights into physician perceptions of unmet needs and expectations for pipeline agents. Further, they have analyzed more than 400 C3G patient charts across regions to reveal the true evolution of treatment patterns and patient types. 

Findings from Spherix’s 2024 Market Dynamix™: Complement 3 Glomerulopathy (US) study that collected perspectives from 101 U.S. nephrologists confirm nephrologists’ strong interest in being able to leverage new therapies for C3G, emphasizing the importance of clinical data that demonstrate slowed eGFR decline, proteinuria reduction, and other critical efficacy markers. They also highlight the value of treatments targeting the complement system pathways to directly address the disease’s underlying mechanisms. 

Insights from Spherix’s Patient Chart Dynamix™: Complement 3 Glomerulopathy (US) study, which analyzed 157 C3G patient records from 94 nephrologists, reveal that physicians would likely treat up to half of their C3G patients with these new agents if approved. At this point, nephrologists project that the majority of audited C3G patients are likely to require dialysis within the next ten years – a rate that surpasses many other renal diseases. This projection underscores their desire and the critical need for treatments that can fill gaps in care and delay disease progression. 

Late-stage candidates from Novartis (Fabhalta) and Apellis (Empaveli) show promise in C3G and are nearing potential FDA approval. Both therapies, already approved in the U.S. for paroxysmal nocturnal hemoglobinuria (PNH), and IgA nephropathy (IgAN) in the case of Fabhalta, are advancing through Phase III trials for C3G and are expected to have regulatory submissions in the coming months. However, Spherix data indicates that treatment preferences are likely to be influenced by the route of administration, with Fabhalta taken orally twice daily and Empaveli administered subcutaneously twice weekly. 

At ASN Kidney Week in October, Novartis presented one-year data from its Phase III APPEAR-C3G trial of Fabhalta (iptacopan), an oral complement inhibitor, on C3G patients. The data demonstrated a significant and sustained reduction in proteinuria within 14 days of treatment, lasting up to 12 months. The drug met its primary endpoint at six months, and Novartis has already submitted applications for approval beyond the U.S. in the EU, China, and Japan. 

Apellis, in collaboration with European partner Sobi, also presented Phase III VALIANT trial data for Empaveli (pegcetacoplan) at the conference, showing a 68.1% reduction in proteinuria versus placebo, with 71.4% of treated patients achieving zero C3c staining intensity, signaling C3c deposit clearance. The VALIANT trial includes patients with C3G and IC-MPGN, demonstrating early efficacy by week four. 

Following ASN, Spherix fielded a follow-up study to U.S. nephrologists who attended the conference to understand their engagement with the meeting content and industry events, sessions that were most popular, reactions to high-impact clinical trials and poster presentations, including the latest updates in C3G, and more. Results will be available this week. 

As the C3G treatment landscape evolves, with increasing investment and promising therapies on the horizon, Spherix will continue to provide in-depth insights into market developments through its Market Dynamix™ and Patient Chart Dynamix™ services, as well as its Launch Dynamix™ tracking series as products gain approval. 

Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents. 

Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns. 

Launch Dynamix™ is an independent service providing monthly benchmarking of newly launched products for the first eighteen months of commercial availability, augmented by a quarterly deep dive into promotional activity, barriers to uptake, and patient types gravitating to the launch brand. 

About Spherix Global Insights   

Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.  

The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.  

As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth.  

To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn.   

For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com  

NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.

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