FDA Treatments

NephCure’s advocates have achieved a significant victory this summer, more than tripling the number of cosponsors for the New Era Act (H.R. 6790). Before NephCure’s Rare Kidneys On The Hill Day event on July 24, the New Era Act had the support of 10 members of Congress—an encouraging start, but a push was needed to gain real traction. Now, thanks to the tireless efforts of our advocacy community, that number has now surged to 37 co-sponsors for the New Era Act as of October 1st, 2024.

This unprecedented increase is a direct result of the dedication of NephCure advocates, who spent the summer contacting and engaging their congressional representatives, sharing their personal stories, discussing the The New Era Act’s impact, and explaining the urgent need for action. From phone calls to emails and face-to-face meetings, these advocates mobilized to make sure their voices — and the needs of the rare kidney disease community — were heard.

Their work demonstrates the profound impact that collective action can have in advancing critical legislation. The New Era Act, which aims to transform the treatment and prevention of end-stage kidney disease, now has the widespread support it needs to move forward, thanks to the persistent and passionate efforts of our community.

While this progress is a major milestone, there is still much work ahead. NephCure remains committed to pushing for the passage of the New Era Act and ensuring that patients with rare kidney diseases receive the attention, care, and support they deserve. 

NephCure is deeply grateful to every advocate who helped make this possible and looks forward to continuing this fight together. Sign up for our monthly newsletter to stay updated as we move closer to making this life-changing legislation a reality!

To see the full list of the most up to date New Era Act co-sponsors, click here.

In June 2024, Reykjavik, Iceland, became the focal point of a significant milestone in the fight against focal segmental glomerulosclerosis (FSGS), a rare kidney disease. The PARASOL Project, launched in December 2023 with the goal of providing data driven and feasible endpoints for the conduct FSGS clinical trials, held a pivotal meeting on June 8-9, 2024, bringing together leading experts and stakeholders from around the globe to assess progress and outline future strategies.  

This marks the PARASOL Project’s second meeting which included researchers, clinicians, patient advocates, industry sponsors, biostatisticians and government regulatory experts. This collective came together to continue the work needed to be done in order to identify alternative endpoints for FSGS clinical trials— particularly considering that the FDA currently accepts complete remission or near normalization of proteinuria as a surrogate endpoint and basis of traditional approval of new treatments. An endpoint is an outcome that can be measured to determine if the treatment being studied is beneficial. 

As more trials are underway or about to begin, there is a need to explore whether lesser changes in proteinuria can be used to support accelerated or traditional approval of new treatments. 

The meeting focused on patient-level data assembled from cohorts and registries from around the globe. To date, more than 3,000 patients are represented from more than 20 cohorts that have committed their data, with four so far, fully integrated into the analysis—NEPTUNE, CureGN, Kidney Research Network (KRN), and UNC Glomerular Disease Collaborative Research Network (GDCRN). Additional cohorts are on track for inclusion pending administrative approvals by mid-August. 

“We are truly inspired by the community’s response to PARASOL, especially the eagerness of nephrologists and their teams to share their data,” PARASOL Project Co-Chair, Dr. Matthias Kretzler said. “Each participating registry or patient cohort often identified additional sources of well-characterized FSGS patients who could potentially join the initiative.” 

The collaborative atmosphere in Reykjavik fostered robust engagement among participants, emphasizing the urgency and potential impact of this work. Biostatisticians Margaret Helmuth, MS, and Abigail Smith, PhD, presented preliminary analyses setting the stage for focused breakout sessions and discussions about additional analyses in preparation to share key data deliverables at the October 2024 annual meeting of the American Society of Nephrology. 

“It was incredibly gratifying to have such a diverse group of stakeholders all in one room, fostering robust and vital discussions,” said Josh Tarnoff, CEO of NephCure. “The range of perspectives—from FDA experts to patient advocates—was invaluable in emphasizing the importance of our shared repository and exploring how to develop an appropriate indicator for FSGS progression in trials to get treatments to those who are in great.” 

The PARASOL Project is pioneering new approaches in the fight against FSGS, creating hope amongst the NephCure community for new, potential treatment options. 

“With an amazing group of experts, we delved into strategies to improve endpoints to better serve patients, clinicians, and sponsors. Our discussions covered practical trial designs, analyzing complex pediatric patient data, and identifying specific patient subgroups to ensure our model holds up,” PARASOL Project Co-Chair, Dr. Laura Mariani explained. 

The PARASOL project is sponsored by NephCure, International Society of Glomerular Diseases, National Kidney Foundation, and Kidney Health Initiative — and most recently, the U.S. Food and Drug Administration (FDA). 

A third PARASOL project meeting will be held in Washington, D.C. on October 7-8, 2024, followed by broader dissemination at American Society of Nephrology Kidney Week later that month in San Diego. 

If you would like to get involved further with the PARASOL project, or have additional questions, please direct all inquiries to  info@nephcure.org.