NephCure, a leading nonprofit organization dedicated to improving the lives of people affected by rare kidney diseases, is proud to recognize IgA Nephropathy (IgAN) Awareness Day on May 14, 2025. This important day is dedicated to elevating awareness of IgA Nephropathy, a rare kidney disease that often progresses without obvious symptoms until significant damage to the kidney has occurred.
NephCure remains committed to empowering patients, caregivers, and healthcare professionals with resources, education, and support to help improve outcomes for those impacted by this condition.
Also known as Berger’s disease, IgA Nephropathy occurs when an antibody called immunoglobulin A (IgA) builds up in the kidneys, causing inflammation that can lead to kidney scarring and eventually kidney failure. For many, IgAN remains undetected for years due to its subtle early symptoms—making awareness and education critical.
Thanks to scientific progress and the dedication of the kidney disease community, new FDA-approved treatment options are now available that can help preserve kidney function and potentially slow disease progression.
Join Us: IgAN Awareness Day Town Hall – May 14 at 7pm ET NephCure will host a free, virtual IgAN Awareness Day Town Hall on Wednesday, May 14 at 7:30 PM ET. This empowering event will include updates from medical experts, lived experiences from patients, and information on navigating treatment options. All are welcome to attend, click here to register.
NephCure extends heartfelt thanks to our generous IgAN Awareness Day sponsors, Novartis, Otsuka, Travere Therapeutics, Alexion, Calliditas Therapeutics, and Vertex Pharmaceuticals, for their continued partnership in raising awareness and supporting the IgAN community.
About NephCure: NephCure’s mission is to empower people with rare, protein-spilling kidney diseases to take charge of their health while leading the revolution in research, new treatments, and care. Since 2000, NephCure has invested more than $40 million in kidney disease research and helped transform the treatment landscape through advocacy, education, and support. NephCure is a U.S. tax-exempt 501(c)(3) public charity.
Philadelphia, PA — May 1, 2025 — NephCure, a leading nonprofit organization dedicated to finding better treatments and a cure for rare, protein-spilling kidney diseases, is proud to announce the launch of its inaugural Advocacy Academy, a four-week program designed to build and strengthen the advocacy skills of rare kidney disease (RKD) patients, families, and allies.
Beginning today, Advocacy Academy participants will embark on a focused journey to become more effective champions for the RKD community. The course will offer targeted training on critical advocacy skills, including legislative engagement, storytelling for impact, and grassroots mobilization.
Graduates of the Advocacy Academy will be fully prepared to participate in Rare Kidneys on the Hill Day on July 23-24, 2025, where they will meet directly with elected officials to advocate for vital policies such as the New Era of Preventing End-Stage Kidney Disease Act (H.R. 1518) and other initiatives supporting RKD patients. Participants will also be empowered to lead advocacy efforts in their own state and local communities year-round.
“Our goal is to equip our advocates with the tools they need to create real change,” said Matthew Johnson, NephCure Director of Government Relations and Advocacy. “By investing in their development, we are strengthening the voice of the RKD community and ensuring policymakers hear directly from those most impacted.”
Throughout the course, participants will engage in interactive sessions, complete quick educational modules via NephCure’s Advocacy Accelerator platform, and work toward a final advocacy project to cap off their training.
NephCure’s Advocacy Academy efforts would not be possible without the generous support of its sponsors, Travere Therapeutics, Novartis, and Otsuka.
For more information about NephCure’s Advocacy Academy or to get involved in rare kidney disease advocacy, please click here or contact advocacy@nephcure.org.
About NephCure
NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
NephCure, a leading nonprofit organization dedicated to finding better treatments and a cure for rare kidney diseases, is proud to announce the second annual APOL1 Kidney Disease Awareness Day, taking place on April 29, 2025. This day is dedicated to raising national awareness of APOL1 kidney disease, which is a genetic form of kidney disease that disproportionately affects people of African ancestry.
APOL1 kidney disease is caused by specific changes in the APOL1 gene, which can lead to an increased risk of developing kidney disease. People who inherit certain APOL1 gene variants may be more likely to develop kidney disease, making education, awareness, and advocacy around this condition critical for early intervention and equitable care.
To honor this important day, NephCure invites everyone to participate in the following awareness opportunities:
Join the Virtual Town Hall on April 29 at 7PM ET NephCure will host a free, live virtual APOL1 Awareness Day Town Hall event featuring powerful perspectives and expert insights from leading voices in the kidney and health equity space, including:
Dr. Keisha Gibson, MD, MPH – A pediatric nephrologist from UNC Chapel Hill.
Corynne Corbett – Representing Black Health Matters.
Barbara Harrison, MS, CGC – A clinical genetic counselor from Harvard University.
Spread Awareness on Social Media Download the toolkit and share NephCure’s APOL1 kidney disease awareness graphics and patient stories on your social media channels, don’t forget to tag @NephCure.
Fast Facts About APOL1 Kidney Disease:
African Americans make up 13% of the U.S. population, but account for nearly 35% of people with kidney failure in the U.S.
1 in 8 African Americans is at risk of a genetic form of kidney disease (caused by the APOL1 gene mutations).
APOL1 kidney disease is particularly aggressive and currently has no FDA-approved treatments.
Approximately 40% of African Americans on dialysis have kidney failure caused by APOL1.
NephCure’s APOL1 Awareness Day efforts would not be possible without the generous support of its sponsors, Vertex Pharmaceuticals, AstraZeneca, and Maze Therapeutics.
To register for the Town Hall on April 29th and learn more about APOL1 awareness efforts, click here.
About NephCure: NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
A major step forward has been made for the IgA Nephropathy (IgAN) community. Last week, the FDA granted accelerated approval to Vanrafia (atrasentan), Novartis’ therapy, for adults with primary IgAN at risk of rapid disease progression. This approval brings new hope to those affected by this progressive, rare kidney disease.
As background, the FDA’s accelerated approval program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need.
IgA Nephropathy (IgAN) is a kidney disease in which Immunoglobulin A (IgA) builds up in the kidney. IgA is a protein in the blood and is also part of the immune system. Excess IgA can cause inflammation in the kidney and over time, this leads to scarring in the kidney tissue. The severity of kidney disease caused by IgAN varies from person to person. As IgAN progresses, it reduces the kidneys’ ability to filter waste from the blood.
Approval was granted based on interim results from Novartis’ Phase 3 ALIGN trial, which demonstrated a 36.1% reduction in proteinuria compared to placebo. Reductions were seen as early as Week 6 and sustained through Week 36. Vanrafia was also shown to have a favorable safety profile, with no new safety signals observed. Ongoing data from the ALIGN study will evaluate whether Vanrafia slows kidney function decline long-term, with final results expected in 2026.
With this latest announcement, there are now four FDA-approved therapies for IgAN — a major shift from just a few years ago:
FABHALTA (iptacopan) – Approved for C3G and IgAN
FILSPARI (sparsentan) – Approved for IgAN
TARPEYO (budesonide) – Approved for IgAN
VANRAFIA (atrasentan) – Approved for IgAN
These treatment options, each with different mechanisms of action, allow patients and their care teams to take a more personalized approach to managing this disease.
NephCure remains committed to supporting the IgAN community through continued education, research, and advocacy. While this is an exciting moment, we know the work is far from over. Our focus now turns to ensuring timely, affordable, and equitable access to these therapies for all who need them.
If you or a loved one has IgAN, we encourage you to speak with your doctor to determine which treatment may be right for you.
To read Novartis’ full press release about Vanrafia’s FDA approval, click here.
For more information on IgA Nephropathy, click here. To view and read the available resources about IgAN, please visit NephCure.org.
A historic milestone has been reached for the rare kidney disease (RKD) community — the FDA has approved the drug FABHALTA as the first-ever treatment option for adults with C3 glomerulopathy (C3G). This long-awaited approval brings new hope to patients and families affected by this devastating disease.
C3G is an ultra-rare, debilitating kidney disease that damages the glomeruli (the filtering units in the kidneys that help remove toxins from the blood) which can ultimately lead to kidney failure. Until now, there were no approved targeted treatment options available for C3G.
“This approval marks a significant step forward in our collective goal to improve the outcomes of those affected by rare kidney diseases. For the first time, the C3G community has a therapy approved and designed specifically for their condition. This comes on the heels of groundbreaking advancements in IgAN, which underscores the current exciting new era of innovation in RKD treatments,” Josh Tarnoff, NephCure’s CEO said.
FABHALTA’s approval for FABHALTA in C3G is based off Novartis’ Phase 3 study, APPEAR-C3G, which evaluated the efficacy and safety of the drug and demonstrated reduction in proteinuria, which was seen as early as 14 days.
This approval marks an important advancement, but our work is far from over. NephCure remains committed to advancing research, advocacy, and patient education to further improve treatment options and outcomes for those with C3G and other rare kidney diseases. With more than 60 clinical trials in the RKD space, we anticipate even more new treatments to be approved in the near future.
Now, the focus shifts to ensuring timely and equitable access to these new and better treatments for all patients.
If you or a loved one has C3G, we encourage you to speak with your doctor to determine if FABHALTA may be an appropriate treatment option for you.
To read Novartis’ press release about FABHALTA’s approval for C3G, click here.
For more information on C3G and available resources, please visit NephCure.org.
PHILADELPHIA, PA – March is Kidney Month, and NephCure is proud to join the global movement to raise awareness about kidney health. As the only kidney organization squarely focused on rare, protein-spiling kidney diseases, NephCure is committed to advocating for the thousands of patients and families affected by conditions such as FSGS, IgAN, Nephrotic Syndrome and other rare kidney diseases.
Though individually classified as rare, these diseases belong to a group of 150 conditions, each affecting fewer than 200,000 people in the U.S. Collectively, rare kidney diseases (RKD) impacts a significant number of individuals, yet they often remain underdiagnosed and misunderstood. NephCure works to change that by providing expert resources, funding groundbreaking research, and helping advance new treatments to improve patient outcomes.
Your Support Changes Lives: NephCure Aims to Raise $25,000 This Kidney Month in Honor of its 25th Anniversary
For 25 years, NephCure has been committed to creating a world where all who are affected by RKD are connected to new and better treatments—and one day, a cure.
As the organization moves towards this milestone, NephCure has set a goal to raise $25,000 during Kidney Month, in honor of their 25th anniversary. Your support directly impacts those affected by RKD, funding critical initiatives such as:
Innovative research to accelerate the discovery of breakthrough treatments
Educational resources to empower patients and families
Patient support programs that connect and uplift the RKD community
Community members can show their support for NephCure throughout Kidney Month by visiting the official Kidney Month landing page. This dedicated resource page offers multiple ways to get involved, including:
Making a donation to fuel critical research and advocacy efforts
Sharing personal stories to help raise awareness of rare kidney diseases
Accessing expert resources to better understand RKD, its impact, and available support
In addition, NephCure has created a social media toolkit to encourage widespread digital advocacy and is offering Kidney Month T-shirts for purchase to help spread awareness.
Any of the actions that can be taken on this page—whether big or small—contribute to the fight against RKD. By engaging with these tools and spreading awareness, individuals can play a vital role in advancing NephCure’s mission.
Corporate Sponsors and Partners Help Drive Awareness and Impact
NephCure’s Kidney Month efforts would not be possible without the generous support of its corporate sponsors: Boehringer Ingelheim, Novartis, Otsuka, and Travere Therapeutics.
Additionally, NephCure is partnering with Xtrema Ceramic Cookware, Black Health Matters, and PateintsLikeMe to amplify the Kidney Month campaign across social media.
Make an Impact This Kidney Month
NephCure invites individuals, organizations, and advocates to join in amplifying the voices of those living with rare kidney diseases. By raising awareness, supporting critical research, and fostering a stronger patient community, we can create a future with better treatments and improved quality of life for those affected.
For more information on how to support NephCure’s Kidney Month initiatives, click here.
About NephCure
NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
PHILADELPHIA, PA – January 31, 2025 – This year marks a monumental milestone for NephCure: 25 years of driving hope, innovation, and progress in the fight against rare kidney disease (RKD).
Founded in 2000 by four families determined to find answers when none existed, NephCure has grown into an international organization, pushing rare kidney disease research and care forward while connecting patients, families, and caregivers to the resources they need.
NephCure has fostered groundbreaking research, advanced awareness, and built a thriving community of advocates, expert physicians, and key stakeholders united in one goal: creating a better world for everyone affected by RKD.
“For 25 years, we’ve made tremendous investments in research and landscape changing initiatives and now we are experiencing remarkable progress that once seemed impossible — groundbreaking research advancements, FDA-approved treatments, and a resilient community determined to find a cure,” said NephCure CEO Josh Tarnoff.
“We’ve gone from having little understanding of these rare kidney diseases to now having multiple treatment options, with more on the horizon. That’s an extraordinary accomplishment in a short time! But this milestone isn’t just about looking back — it’s about harnessing our momentum to drive even greater breakthroughs. Once we could only offer hope. We are now in an era where we can offer real options that may stop disease progression.”
“We’ve gone from essentially knowing nothing about these rare kidney diseases to having several treatments options available, and more in the pipeline. That’s an incredible achievement in a relatively short amount of time! However, this anniversary isn’t just about celebrating how far we’ve come; it’s about rallying our collective energy to keep pushing forward.”
NephCure’s 25-year journey is marked by transformative achievements, including:
Supporting the NIH’s first clinical study for FSGS patients in 2001, which laid the foundation for understanding the disease today.
Co-funding the NEPTUNE study in 2009, an effort that provided crucial insights for researchers into nephrotic syndrome.
Launching the Kidney Health Gateway in 2019, (now our Clinical Trials page) giving those affected by RKD access to clinical trials and the latest treatment options.
Leading advocacy efforts, resulting in unprecedented attention from policymakers and an increase in federal research funding.
These milestones underscore NephCure’s relentless pursuit of a future where RKD patients no longer face uncertainty or isolation.
Although there is much to celebrate, the work is far from over. With FDA approvals for breakthrough treatments on the horizon and exponential momentum in RKD research, the next 25 years hold even greater promise. NephCure is committed to expanding its impact — empowering the next generation of nephrologists, fostering collaborations across industries, and ensuring no patient or family walks this journey alone.
As part of its 25th anniversary celebration, NephCure invites everyone to explore its visual history, milestones, and future goals at NephCure.org/25th-Anniversary. Together, we can honor the progress made and continue driving meaningful change for those affected by rare kidney disease.
About NephCure
NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
Honored for NephCure’s APOL1 Kidney Disease Awareness Campaign
KING OF PRUSSIA, Pa. (Nov. 19, 2024) – NephCure is honored to announce its Bronze-level win at the 4th Annual Anthem Awards in the Health category. This recognition highlights the impactful work of NephCure’s APOL1 Kidney Disease Awareness Campaign, a groundbreaking initiative dedicated to raising awareness of this genetic condition predominantly affecting the Black community.
The Anthem Awards, presented by the International Academy of Digital Arts and Sciences, celebrate outstanding achievements in social impact and purpose-driven work. This year’s competition was the most competitive yet, with over 2,300 entries submitted from more than 30 countries. Additionally, over 33,000 supporters cast 47,000 votes for finalists in the Anthem Community Voice Awards. NephCure’s win underscores its commitment to advancing health equity and awareness on a global scale.
Patricia McLoughlin, General Manager of the Anthem Awards, praised this year’s honorees, saying, “The winners of this year’s Anthem Awards are truly inspiring, and I am honored to help elevate their impact. At a time of uncertainty, the tireless efforts of the Anthem Awards community provide hope for a better tomorrow.”
The success of this initiative is credited to the dedicated NephCure team, including Kylie Karley, Director of Marketing & Communications; Imanté Eichelberger, Coordinator of Marketing & Communications; Destiny Lalonde, Content Marketing Manager; Montrez Lucas, Associate Director of Patient Navigation; and Maurice Madden, Director of Stakeholder Engagement. These leaders will accept the award on behalf of NephCure, celebrating their collective efforts to make a meaningful impact.
About APOL1 Kidney Disease Awareness:
APOL1 kidney disease is caused by genetic variations in both copies of the APOL1 gene, significantly increasing an individual’s risk of developing kidney disease—by as much as 10 to 30 times. These gene changes occur exclusively in people of African descent, including those who identify as Black, African American, Hispanic, Latino, or Afro-Caribbean. NephCure’s campaign aimed to empower these communities with tools for early detection, education, and preventative care, fostering greater understanding and action.
Campaign Impact:
NephCure’s award-winning campaign was anchored by the inaugural National APOL1 Kidney Disease Awareness Day, the first of its kind. The campaign sought to bring attention to the increased risk of kidney disease among individuals of African descent due to genetic variations in the APOL1 gene.
Through partnerships with more than ten community organizations, including historically Black colleges and universities (HBCUs) and faith-based groups, the campaign brought kidney health awareness directly into the communities most affected. Over 300 individuals were screened for kidney disease during events held across the country. Attendees received educational materials, and more than 100 free T-shirts were distributed to encourage participation and engagement.
The campaign extended its outreach through email marketing efforts, which saw significant engagement. A “Save the Date” email was delivered to 2,929 recipients, achieving a 30.26% open rate and a click-through rate (CTR) of 2.9%. A follow-up email dedicated to APOL1 Day reached 2,993 recipients, with a 24.17% open rate and a CTR of 2.8%.
A virtual component of the campaign further amplified its reach. The first APOL1 webinar attracted 100 registrants, with 55 concurrent viewers and 93 total viewers through NephCure’s platform. Additionally, the event was streamed via Soulivity’s website, where it garnered over 89,000 viewers. The APOL1 Day webpage saw a 21% conversion rate, further illustrating the campaign’s effectiveness in inspiring action and awareness.
About NephCure:
NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. As NephCure continues its mission, it remains dedicated to setting new benchmarks in health equity, creating opportunities for early intervention, and offering hope to communities in need. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now more than 60 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
Looking Ahead:
This Bronze Anthem Award reinforces NephCure’s commitment to improving the lives of individuals at risk for and living with rare kidney diseases. The APOL1 Kidney Disease Awareness Campaign serves as a powerful example of how community-driven efforts and purposeful partnerships can drive meaningful change.
For more information about APOL1 Kidney Disease or to support NephCure’s work, please visit https://nephcure.org/.
BETHESDA, MD. October 14, 2024 – A multi-stakeholder group of rare kidney disease experts aligned around a potential proteinuria-based clinical trial endpoint, balancing biological relevance and trial design considerations.
PARASOL (Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis [FSGS]), an initiative co-sponsored by NephCure, the International Society of Glomerular Disease, the U.S. Food and Drug Administration, the Kidney Health Initiative, and the National Kidney Foundation, convened for the third time in the past ten months at a public scientific workshop held in Bethesda, Maryland on October 7-8, 2024. FSGS is an important cause of kidney failure in patients of all ages and new therapies are urgently needed to reduce the risk of progression. The objective of PARASOL is to advance the understanding and use of proteinuria and eGFR-based surrogate endpoints for accelerated and traditional approval of new treatments for FSGS, thus facilitating the development of new therapies. PARASOL represents the collaborative effort of nephrologists, scientists, patient advocates, biostatisticians, trial sponsors, and regulatory authorities.
At the meeting, the biostatistical team reported an in-depth analysis of 1626 children and adults with FSGS from a combined dataset drawn from multiple glomerular disease networks around the world. Analysis lead Abigail Smith, PhD, Associate Professor of Biostatistics, Department of Preventive Medicine, Northwestern University Feinberg School of Medicine, reported: “The principal finding is that reduction in proteinuria over 24 months is strongly associated with a reduction in the risk of kidney failure, and responder definitions based on thresholds of proteinuria are both biologically plausible and strongly supported by epidemiological data. The finding was consistent in subgroups based on patient age, level of baseline proteinuria, or presence of chronic kidney disease and was validated in a large independent European patient cohort.” Discussion of the findings in an open forum highlighted their broad utility, the biological role of proteinuria in FSGS as a podocytopathy, and implications for clinical trial design.
“PARASOL is an incredible demonstration by the international nephrology community that through generosity and multidisciplinary collaboration, we can answer fundamental, practical scientific questions needed to inform clinical trial design in rare disease,” said Dr. Laura Mariani, PARASOL Co-Chair and Associate Professor of Medicine (Nephrology) at the University of Michigan.
Laurel Damashek, Executive Director of the International Society of Glomerular Disease, commented, “In under a year, we have taken a giant step forward for FSGS thanks to the dedication and expertise of this collaborative workgroup. We will continue to integrate datasets from additional entities that have generously committed to sharing. Our team hopes to maintain and leverage the PARASOL infrastructure to address urgent questions in other glomerular diseases. PARASOL has shown that sharing data is vital to our collective ability to advance glomerular medicine and improve the lives of people living with rare kidney diseases like FSGS.”
The results of PARASOL will be presented to the full nephrology community on October 25, 2024 at a dedicated session of the annual Kidney Week meeting of the American Society of Nephrology in San Diego, California. Plans for publication in a peer-reviewed journal are also underway.
About PARASOL: PARASOL is a collaborative project sponsored by the U.S. Food and Drug Administration, NephCure, the International Society of Glomerular Disease, the Kidney Health Initiative of the American Society of Nephrology, and the National Kidney Foundation. It was launched in December 2023 to advance the understanding and use of proteinuria and eGFR-based endpoints as surrogate endpoints for accelerated and traditional approval in FSGS by facilitating new analyses of existing data from randomized controlled trials, observational studies, and registries.
About NephCure: NephCure is the only organization committed exclusively to accelerating research, education, and support to improve outcomes for individuals living with rare, protein-spilling kidney diseases like Focal Segmental Glomerulosclerosis (FSGS) and Nephrotic Syndrome. NephCure works to connect patients to clinical trials, advances research for new treatments, and advocates for policies that benefit those with kidney diseases.
October 7, 2024: – NephCure is excited to announce the official launch of their new online shop, offering a range of branded merchandise to help continue to raise awareness and support the rare kidney disease (RKD) community. NephCure’s new online store features a variety of items including t-shirts, hoodies, and sweatshirts for both adults and children, designed to help individuals show their support for the (RKD) community and to help create a sense of unity and togetherness.
All proceeds from the NephCure Shop will go directly to the organization — helping fulfill the organization’s mission to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Those who purchase from the NephCure Shop will directly help fund innovative research initiatives, patient education programs, and advocacy efforts aimed at improving patient outcomes and finding a cure.
By purchasing from the NephCure Shop, individuals will not only help spread awareness of RKD, but also provide crucial funding to ensure continued advancements in treatment options and care.
To explore the shop and show your support for the kidney disease community, visit: NephCure.org/shop.
About NephCure:
NephCure is the only organization committed exclusively to accelerating research, education, and support to improve outcomes for individuals living with rare, protein-spilling kidney diseases like Focal Segmental Glomerulosclerosis (FSGS) and Nephrotic Syndrome. NephCure works to connect patients to clinical trials, advances research for new treatments, and advocates for policies that benefit those with kidney diseases.
September 3, 2024: – NephCure, a leading nonprofit organization dedicated to finding better treatments and a cure for rare kidney disease, is proud to announce the inaugural IgA Nephropathy (IgAN) Awareness Day on September 26, 2024. The event is specifically dedicated to raising awareness about IgA Nephropathy, a rare and often “silent” kidney disease.
This day marks a significant milestone for the IgAN community, as NephCure aims to shine a spotlight on this under recognized condition, promote the importance of early diagnosis and proactive treatment, and provide crucial resources for those affected.
IgAN, also known as Berger’s disease, is a kidney condition that causes inflammation in the kidney’s filtering units, potentially leading to kidney damage and even kidney failure. For many years, IgAN has been a silent adversary, often progressing undetected due to a lack of recognizable symptoms. However, the landscape of IgAN treatment is changing, as new FDA-approved treatment options are now available, offering hope to patients by helping preserve kidney function and potentially delaying or preventing the need for dialysis or transplant.
Understanding the IgAN Journey To coincide with IgAN Awareness Day, NephCure is launching a powerful digital campaign titled “Silent but Serious: Understanding IgA Nephropathy.” This campaign will feature compelling stories from four IgAN patients who have navigated the challenges of this condition, as well as several different educational resources and support options.
Join Us for the IgAN Awareness Day Town Hall As part of the awareness day events on September 26th, NephCure will host a free, virtual IgAN Awareness Day Town Hall at 7:30 pm EDT. This insightful and empowering event will bring together patients, caregivers, and medical professionals to discuss the latest advancements in IgAN treatment, share patient testimonials, and provide information about the newly launched IgAN Patient Assistance Program. We invite everyone impacted by IgAN to join this important conversation. To register for the webinar, click here.
Why IgAN Awareness Matters IgA Nephropathy often goes undetected in its early stages, with up to 40% of patients showing no recognizable symptoms at the time of diagnosis. Despite its slow progression, there is a growing urgency to treat IgAN, as even patients with lower levels of protein in their urine (proteinuria) are at risk of progressing to end-stage kidney disease (ESKD). NephCure is dedicated to providing the tools and resources needed for patients to take charge of their health, including the newly released IgAN Patient Handbook, which offers a comprehensive guide to understanding and managing the disease.
The IgAN Patient Handbook also provides practical advice on managing daily life with IgAN, such as valuable dietary tips, lifestyle adjustments, and effective communication strategies with healthcare providers. To download this resource, click here.
NephCure thanks our generous sponsors, Travere Therapeutics, Alexion, and Calliditas Therapeutics for supporting this event and making IgAN Awareness Day possible.
For more information about IgAN Awareness Day, to register for the town hall, or to download the IgAN Patient Handbook, click here.
About NephCure: NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
August 8, 2024: Today marks a promising day for the rare kidney disease (RKD) community: the FDA has announced that Novartis’ drug, Fabhalta (iptacopan), has been granted accelerated approval for the treatment of IgA nephropathy in adults.
As background, the FDA’s accelerated approval program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need. Fabhalta is granted accelerated approval based on reduction of proteinuria. Further evaluations will determine if the drug slows kidney function decline in IgAN patients.
Over the next several months, Novartis will share more information about this new treatment and explain when and how those who need the drug can access it.
“The FDA’s accelerated approval of Fabhalta represents a critical advancement in addressing the serious and debilitating symptoms of IgA nephropathy. It adds another key option for the rare kidney disease community, as patients respond differently to various therapeutic agents, and we need more options offering different mechanisms of action,” said Josh Tarnoff, NephCure’s CEO.
“This is a momentous occasion that underscores the significant progress our community continues to make in developing effective treatments for this devastating disease. We know that our work is far from over, but these breakthroughs are critical to ensuring our patient community has increasing access to life-saving treatments. This is not just hope – this is a path paved forward.”
The continued approval of Fabhalta may be dependent upon the data from Novartis’ ongoing phase 3 APPLAUSE-IgAN study, which evaluates whether Fabhalta slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline over 2 years. The eGFR data are expected at study completion in 2025 and are intended to support traditional FDA approval.
Key takeaways regarding Fabhalta:
Fabhalta achieved a 44% proteinuria reduction from baseline in Phase III APPLAUSE-IgAN interim analysis, compared with 9% in placebo arm, demonstrating a clinically meaningful reduction of 38% vs. placebo.
Fabhalta is an inhibitor of the alternative complement pathway, activation of which is thought to contribute to the pathogenesis of IgAN.
Despite current standard of care, up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis.
This marks the first approval from Novartis’ renal pipeline, which also includes atrasentan and zigakibart.
Fabhalta is also being developed to treat several other rare diseases beyond IgA nephropathy (IgAN), such as C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN). Ongoing studies are assessing its safety and effectiveness for these conditions to support potential regulatory approvals. The company plans to submit Fabhalta to the FDA and EMA for C3G treatment by the end of the year.
While we celebrate the FDA’s accelerated approval of Fabhalta, we also continue to move forward to ensure continued access to treatments like this. Our focus now turns to ensuring all patients have timely and equitable access to this new and promising treatment.
NephCure remains steadfast in our mission to empower people with protein-spilling kidney conditions to take charge of their health, while leading the revolution in research, new treatments, and care.
We are deeply grateful for the incredible support that has helped our community reach this pivotal moment, and we are more committed than ever to the important work ahead. There is still much to be done, but today’s news gives us hope and determination to deliver on our promise to the rare kidney disease community.
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