Press Release Archives

NephCure Celebrates First-Ever C3G Awareness Day on September 25, 2025

NephCure, a leading nonprofit organization dedicated to improving the lives of people affected by rare kidney diseases, is proud to recognize the inaugural C3G Awareness Day on September 25, 2025. This day is dedicated to raising awareness and understanding of C3 glomerulopathy (C3G), an ultra-rare kidney disease that can progress to kidney failure.

C3G Awareness Day aims to elevate patient and caregiver voices, provide education about this complex disease, and bring together the broader rare kidney community.

C3G is caused by abnormal activity in the complement system, a part of the immune system that helps the body fight infections. When the complement system is overactive, it can attack the kidneys, leading to inflammation, scarring, and progressive kidney damage. C3G affects people of all ages, but because it is so rare and difficult to diagnose, many patients spend years searching for answers before finding the right care.

This year, the C3G community has seen important progress with the approval of two FDA-approved treatment options; FABHALTA and EMPAVELI. While more work remains to improve access and develop additional therapies, these milestones bring new hope to those impacted by C3G.

NephCure has launched a dedicated C3G Awareness Day webpage featuring educational resources, patient stories, and tools to help spread awareness. There, users can also find a downloadable social media toolkit, which includes ready-to-use graphics and captions. The toolkit allows patients, caregivers, friends, and supporters to share stories and messages of solidarity, whether they are directly affected by C3G or another rare kidney disease.

We thank our partners Apellis, Novartis, Sobi, CompCure, and C3G Warriors, for their commitment to improving the lives of C3G patients and families.

To learn more about C3G Awareness Day, read patient stories, or download the toolkit, visit NephCure.org/c3g-awareness-day-2025.

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.

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Get College Ready! 5 Things Every RKD High School Senior Should Know

Starting college is a major life transition and for students living with rare kidney disease (RKD), it comes with added layers of complexity. Whether you’re preparing to leave home for the first time or supporting a young adult who is, it can be tough to know where to begin.

To help support young adults and families through this life stage, NephCure spoke with Sean Converse, a college senior and a young adult with RKD, who offered valuable insight into what it really means to prepare for the transition to college while managing rare kidney disease.

1. Starting the Conversation at Home
Before leaving for college, Sean emphasized the importance of having an open and honest conversation between teens and parents about health responsibilities.

“Outline expectations around diet, medication management, and mental health care. Once these expectations are outlined and agreed upon by both the parent and teen, they need to work to build trust that the teen can fulfill those responsibilities,” said Sean.

2. Choosing the Right Campus
When considering where to attend college, Sean recommends factoring in healthcare access and available on-campus resources.

Look into how close the campus is to nephrologists or other specialists. If the campus is far away from nephrology care, it will add stress to student and family. If you need help finding a specialist in your area, NephCure can help point you in the right direction. Please click the button below and fill out the form to receive assistance.

Patient Navigation Services

Make sure that the university has a disability services office, sometimes called the Academic resource Center (ARC), or 504 Office, that will help you secure documented accommodations.

Sean said, “When you go on the campus tour, ask about what resources they offer for someone with a rare disease. Then, work with the resource center and your doctor to have accommodation documents drafted before school starts. Make sure that everything is documented, being very detailed in what is allowed and not allowed and get a copy of the documents.”

Examples of accommodations might include extra time on exams, excused absences for medical appointments, dietary modifications in dining halls, or access to a private room or bathroom.

It is extremely important that students with rare kidney disease take the initiative to get their accommodations formalized through the university. This is because professors cannot require students to disclose private health information unless the student chooses to share it through the official accommodations process.

FERPA (the Family Educational Rights and Privacy Act) protects the privacy of students’ education records. It does not prevent a professor from casually asking a student how they are doing. For example, a teacher may ask, “Are you feeling okay?” or “Is everything alright?”—that is not a FERPA violation. However, students are never obligated to share medical details, and professors are not allowed to demand or keep records of health information outside of the formal accommodation process. So, if the accommodations and details of the student’s medical struggles are not documented, a professor may never fully understand the student’s needs.

“By having the conversations to confirm that you will have access to resources, you put yourself in the best position to succeed,” said Sean.

Sean shared that students could receive accommodations regardless of school size. “You can get accommodations in a smaller school or a big school. I go to a small school, and I got the accommodations I need. On the other hand, I helped my friend Ella, who has a rare kidney disease, have a car on campus, when that normally is not allowed, and get a private room.”

3. Dorm Life and Roommate Selection
Sean stressed that every student’s preference and medical needs are different when it comes to dorm life. Some may prefer a private room; others may opt for traditional shared housing. What matters is that students feel comfortable and understand what their options are.

Roommate selection can be approached however feels right. Students don’t have to disclose their diagnosis unless or until they feel ready.

4. Preparing Accommodations During Senior Year
To start college strong, Sean recommends using senior year of high school to proactively prepare. This includes:

Gathering documentation for accommodation requests.

Reaching out to the university’s disability services office early to understand the process.

Speaking with a medical team to determine whether living away from home is appropriate. “Ask if moving out is something they recommend? A simple question may lead to a broader conversation,” said Sean.

5. Preparing Emotionally and Socially
Finally, Sean reflected on the social side of the transition. Moving into a new peer group can be daunting, especially for young adults who have previously felt misunderstood or isolated due to their health.

His advice is simple but meaningful: be open to meeting people and share your diagnosis only when and if you feel ready. Building friendships with people who are accepting and respectful can make a big difference during this transitional period.

“Be yourself, don’t be afraid to put yourself out there. Be willing to go out and meet people. You don’t have to mention your disease at first if you don’t want to. And be friends with people that accept you,” said Sean.

You’ve got this!
Preparing for college as a student with a rare kidney disease involves more than just packing and registering for classes. It’s about developing independence, advocating for support, and setting yourself up for success both academically and medically.

With insights from peers like Sean and the support of NephCure’s tools and community, students and their families don’t have to navigate this transition alone.

For more guidance and to connect with others in the rare kidney disease community, visit the Get Involved tab on our website.

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NephCure Advocates Make Massive Impact on Capitol Hill at 2025 Rare Kidneys on the Hill Day

Washington, DC – August 19, 2025 – On July 23–24th, 2025, NephCure hosted its largest-ever Rare Kidneys on the Hill Day event that brought together 78 advocates from across 26 states, to raise awareness and educate lawmakers and advance legislative solutions for the rare kidney disease (RKD) community.

The advocacy event focused on building support for H.R.1518, the New Era of Preventing End-Stage Kidney Disease Act (New Era Act), the only bipartisan legislation solely focused on RKD that was reintroduced during Rare Disease Week by Representatives Gus Bilirakis (R-FL) and Terri Sewell (D-AL). This landmark legislation aims to improve education and research, develop better diagnostic tools and effective treatments to delay kidney failure for individuals living with rare, protein-spilling kidney diseases like FSGS, IgAN, and minimal change disease. The bill would establish NIH Centers of Excellence to advance rare kidney disease research, expand early diagnosis through genetic testing, and improve care particularly in underserved communities. In addition, it calls for creating nephrology fellowships and primary care physician training to better diagnosis RKD and prevent long diagnostic journeys for patients. It also directs HHS to study treatments that could reduce the need for dialysis and transplants, with advocates urging Congress to maintain critical NIH funding to protect progress.

Reflecting on the importance of the event, Matt Johnson, NephCure’s Director of Government Relations and Advocacy, emphasized:

“Rare Kidneys on the Hill Day is about making sure the voices of patients and families are heard by policymakers in the halls of Congress. For far too long, rare kidney diseases have been overlooked in research funding, treatment development, and public awareness. This year, nearly 80 advocates met with 116 congressional offices to share their stories, push for critical legislation like the New Era Act, and ensure that rare kidney disease is a priority. We are incredibly grateful to the dedicated advocates who participated and it’s their courage and commitment that are the driving force behind NephCure.”

The event kicked off with a Rally Dinner the night before the meetings on the Hill, where participants connected over shared experiences and prepared to share their personal journeys with lawmakers. Nearly two-thirds of attendees were first-timers, and many came with stories of long diagnostic delays, limited treatment options, and emotional tolls of navigating complex conditions.

Over the course of two days, the NephCure community held 116 total meetings with congressional offices including 49 Senate, 67 House, and one direct Member-level meeting. These meetings spanned 26 states and included representation from 65 Democratic, 50 Republican, and one Independent office. In these meetings, advocates introduced themselves, shared their personal experiences with rare kidney disease, and explained how the New Era Act would directly impact their lives or the lives of their loved ones. These stories helped put a human face to the policy, reinforcing the urgency and need for congressional support. Their advocacy directly resulted in 13 new co-sponsors signing onto the New Era Act, bringing the total to 33 co-sponsors as of August 18th, 2025.

This progress would not have been possible without the collective effort of the RKD community and the support of our partners. NephCure extends its gratitude to our generous sponsors: Travere Therapeutics, Novartis, Otsuka and Vertex.

If you weren’t able to attend Hill Day, you can still make a difference in NephCure’s advocacy efforts. Contact your Members of Congress and urge them to support the New Era Act, legislation that will advance critical research, improve patient outcomes, and bring us closer to better treatments for rare kidney diseases. Your voice matters and together, we can help ensure patients and families have the resources and hope they deserve.

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.

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NephCure Launches Improved Clinical Trial Page to Help Rare Kidney Disease Patients Find Studies That Match Their Unique Health Needs

Aug. 1st, 2025 – NephCure proudly announces the launch of its redesigned Clinical Trials webpage and Am I a Match? tool, built to offer an easier and more personalized way for patients and caregivers to search for clinical trial opportunities for rare, protein-spilling kidney diseases.

These updates are designed with the rare kidney disease (RKD) community at the center, aiming to reduce confusion, save time, and help people feel more confident navigating clinical trials. By simplifying how patients find and understand research opportunities, this launch reflects NephCure’s ongoing goal of connecting people living with RKD to expert care as quickly as possible.

What’s New?

At the core of the update is the improved “Am I A Match?” tool – an interactive questionnaire that helps patients quickly see which clinical trials might be a good fit for them based on their current health situation.

The tool focuses on a few non-identifying questions about things like:

Your RKD diagnosis
If you’re on dialysis or had a transplant
Whether you’ve had a kidney biopsy
Your kidney function, and past treatments.

Once the questionnaire is completed, the tool filters available clinical trials based on the responses, so users can see only the studies that might apply to them.

Additional filtering features allow users to:

Filter results by age group, trial type, diagnosis, enrollment status, and location
Search by zip code to find trials close to home
Download a personalized list of trial matches as a PDF to share with their doctor, care team, or family

Each clinical trial listing gives users a quick snapshot of what the study is about, who it’s for, and what’s involved. If individuals find a trial that seems like a good fit, they can fill out a form and click “I’m Interested” to directly send an inquiry to the research team.

For many living with rare, protein-spilling kidney diseases like FSGS, IgAN, or C3G, clinical trials can provide early access to promising treatments. This updates webpage is designed to make the process of understanding which clinical trials are relevant to be less intimidating and more accessible.

To explore the redesigned Clinical Trial webpage and “Am I a Match?” tool, visit https://nephcure.org/managing-rkd/clinical-trials/.

About NephCure:

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The FDA Approves EMPAVELI, New Treatment Option for C3G and Primary IC-MPGN

In a continued wave of progress for rare kidney disease (RKD) treatments, the FDA has approved the drug EMPAVELI (pegcetacoplan) for use in patients 12 and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). This approval also includes individuals who have experienced disease recurrence after kidney transplant.

C3G and primary IC-MPGN are ultra-rare, progressive diseases that damage the filtering units of the kidneys and can ultimately lead to kidney failure.

“The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,” said Josh Tarnoff, NephCure CEO. “We recognize Apellis’ commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.”

What the Research Shows

The FDA’s approval of EMPAVELI is based on results from a Phase 3 clinical trial called VALIANT, which looked at how well the treatment worked in people with C3G or primary IC-MPGN.

The study found that patients who received EMPAVELI experienced:

A 68% reduction in proteinuria, a key sign of disease activity
Stabilization of kidney function, helping to slow disease progression
Significant reduction in C3 deposits in the kidneys, with 71% of patients showing no remaining deposits on follow-up testing

What This Means for the RKD Community

With this approval, EMPAVELI becomes an additional option for patients and families navigating life with C3G or primary IC-MPGN. It follows the FDA’s March 2025 approval of FABHALTA for adults with C3G and continues the encouraging trend toward increased treatment options for people with rare kidney diseases.

There are now five FDA-approved treatment options for rare kidney disease:

EMPAVELI (pegcetacoplan) – Approved for C3G and Primary IC-MPGN
FABHALTA (iptacopan) – Approved for C3G and IgAN
FILSPARI (sparsentan) – Approved for IgAN
TARPEYO (budesonide) – Approved for IgAN
VANRAFIA (atrasentan) – Approved for IgAN

NephCure remains dedicated to advancing education, support, and advocacy for individuals affected by C3G and other RKDs. The organization encourages patients and families to speak with their healthcare providers to determine whether EMPAVELI or any other available treatment may be appropriate based on individual needs.

To read Apellis’ full press release on EMPAVELI’s approval, click here.

For more information on C3G, IC-MPGN, and available resources, visit NephCure.org.

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NephCure Celebrates Inaugural FSGS Awareness Day on June 10, 2025

NephCure, a leading nonprofit organization dedicated to improving the lives of people affected by rare kidney diseases, is proud to recognize the first-ever FSGS Awareness Day on June 10, 2025. This landmark day is dedicated to shining a light on focal segmental glomerulosclerosis (FSGS), a rare and progressive kidney disease that affects all age groups and often leads to kidney failure.

FSGS Awareness Day aims to elevate the voices of patients and caregivers, spotlight the urgent need for earlier diagnoses and better treatment options, and honor the strength of the community navigating this disease every day.

FSGS is characterized by scarring in the kidney’s filtering units, which leads to protein loss in the urine and progressive loss of kidney function. It is a leading cause of end-stage kidney disease in children. Despite its serious impact, FSGS remains largely unfamiliar to the public — making education and advocacy critically important.

NephCure is committed to providing support and educational resources for those affected, while accelerating research and access to care. With continued collaboration between patients, healthcare providers, researchers, and industry, progress is being made toward treatments that can slow progression and improve quality of life.

Join Us: FSGS Awareness Day Town Hall – June 10 at 7pm ET

To mark this important occasion, NephCure will host a free, virtual FSGS Awareness Day Town Hall on Tuesday, June 10 at 7pm ET. The event will feature insights from leading nephrologists Dr. Kirk Campbell and Dr. Rasheed Rastogi, alongside the powerful story of Jamayka Webb, an individual living with FSGS. Together, they will discuss the current landscape of FSGS care, emerging treatments, and how patients and families can advocate for themselves. Click here to register.

Thank you to our corporate partners, Travere Therapeutics, Boehringer Ingelheim, Dimerix, Sanofi, and Apellis, who are working to improve the lives of FSGS patients and families.

To learn more about FSGS Awareness Day, register for the Town Hall, or explore patient resources, visit NephCure.org/fsgs-awareness-day-2025.

Thank you to our corporate partners

Those that are working to improve the lives of FSGS patients and families.

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NephCure Launches Advocacy Academy to Empower Rare Kidney Disease Advocates

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, Otsuka, and Vertex.

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

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NephCure to Recognize IgA Nephropathy (IgAN) Awareness Day on May 14, 2025

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.

To learn more about IgAN Awareness Day, register for the Town Hall, or explore patient resources, visit NephCure.org/igan-awareness-day-2025

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NephCure Celebrates Second Annual APOL1 Kidney Disease Awareness Day on April 29, 2025

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 29^th 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.

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The FDA Grants Accelerated Approval to Vanrafia, New Treatment Option for Adults with IgA Nephropathy

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.

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The FDA Approves FABHALTA, First Treatment Option for Adults with C3G

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 marks the third FDA approval, in the U.S., for FABHALTA and its second within the Novartis’ kidney disease portfolio in less than a year. In August 2024, the drug received accelerated approval for IgA Nephropathy (IgAN).

A New Era of Kidney Care

With this latest breakthrough, there are now four FDA-approved therapies for rare kidney disease:

FABHALTA (iptacopan) – Approved for C3G and IgAN
FILSPARI (sparsentan) – Approved for IgAN
TARPEYO (budesonide) – Approved for IgAN

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.

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NephCure Celebrates Kidney Month this March with Awareness and Fundraising Campaign

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 25^th 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 25^th 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.

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