NBA Hall-of-Famer Alonzo Mourning Shares Personal Story to Raise Kidney Disease Awareness November 18, 2021 by Kylie Winkler 1 Comment KING OF PRUSSIA (Nov. 18, 2021) – NBA hall-of-famer Alonzo Mourning was at the height of his basketball career—he had just won gold in the 2000 Summer Olympics when he noticed extreme swelling throughout his body and a lack of energy. A routine physical exam showed abnormalities, and eventually, Mourning received a diagnosis of a rare, protein-spilling kidney disease called focal segmental glomerulosclerosis (FSGS). “I said ‘Doc, am I going to die?’ He paused; he took too long to answer me. And he said, ‘We have no known cure’… ultimately, he said, ‘In about 10-12 months you’ll probably be on dialysis,’” Mourning said as he recalled the jarring day. Mourning, who missed the entire 2002-2003 season due to kidney disease and later went on to win the World Championship with the Miami Heat in 2006, publicly recounted his personal journey with FSGS in a video for NephCure Kidney International’s kidney disease awareness campaign, debuting Nov. 18. In highlighting Mourning’s story, NephCure aims to reach those who are at risk for kidney disease and to educate the public about the signs and symptoms of these conditions. Black Americans are 4-5 times more likely to develop kidney failure than white Americans, and 1 in 8 are at risk for a genetic form of kidney disease. A variation on the APOL1 gene contributes to this disparity. “We are proud to work with Alonzo Mourning in sharing such an important message that will inspire and inform others who fall within this at-risk population. Our goal is to find a cure for this debilitating kidney disease, as well as educate and support those who are affected by it,” said Michael Levine, NephCure Board President and kidney disease patient parent. This awareness campaign comes just months after NephCure launched its Health Equity Initiative, with the goal of ensuring equitable access to advancements in research, treatments, and care, and to reach individuals from communities of color earlier in their disease progression, preventing or delaying their need for dialysis and transplantation. Support for this video and the awareness campaign was provided by Vertex Pharmaceuticals, Travere Therapeutics, and other funders of the Health Equity Initiative. To watch the entire video with Alonzo Mourning, click here. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. 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 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. Learn more at NephCure.org. ###
The Eyes Behind the Microscope: Meet Dr. Astrid Weins September 30, 2021 by Kylie Winkler Astrid Weins, MD, PhD, is an Associate Pathologist at Brigham and Women’s Hospital in Boston, MA, and an Assistant Professor of Pathology at Harvard Medical School. Her research focuses on understanding acute Nephrotic Syndrome and podocyte injury. She does this by combining observations with state-of-the-art imaging and tissue interrogation techniques. Learn more about her work on Minimal Change Disease (MCD) and about the TRACTION-2 trial, Goldfinch Bio’s clinical research trial investigating GFB-887 with the hope of preventing the progression of kidney disease in patients with treatment-resistant Minimal Change Disease, FSGS, and Diabetic Nephropathy. The TRACTION-2 trial is the first large-scale clinical trial opportunity for Minimal Change Disease patients, and is also available for patients with FSGS and Diabetic Nephropathy. Why are you interested in Minimal Change Disease (MCD)? Astrid Weins, MD, PhD I have studied kidney, and specifically, podocyte biology for many years. The podocyte is a unique and fascinating cell with a critical function in regulating urine filtration. It is the main target of injury in MCD. As a kidney pathologist, I love scientific investigation, and my strong belief is that knowing the cause of a disease is the key to its cure. Minimal Change Disease deserves so much more attention than is has received in the past. People often refer to it as a “benign” condition, because in many cases it responds well and fast to steroids. However, there are some children, and even more adults that don’t respond to this therapy, and we have currently no way of predicting who will respond and who won’t. Once we understand what causes a disease, we can develop rationales for choosing and developing specific, targeted therapies. What makes MCD different from other protein-spilling kidney diseases? First, it’s of rapid onset and in most cases also shows a rapid response to therapy. Second, we have had no idea what causes it, other than that we presume it’s caused by a circulating factor. Third, in contrast to most other protein-spilling diseases, it is more common in children than in adults. Fourth, the kidney biopsy shows no significant changes by light microscopy, although the ultrastructural changes to the podocytes are widespread and severe. Hence, the term “minimal change” disease (which refers to its unremarkable light microscopy appearance) is really a misnomer. Can you summarize your research on MCD and its significance? In our clinical practice, my colleagues and I came to recognize a unique feature in biopsies from patients with MCD, which suggested that the injury might be caused by an autoantibody against podocytes. An autoantibody-mediated cause would make sense, since antibodies are circulating in the blood, are filtered by the kidney and can access the podocytes. We hypothesized that the target of such an autoantibody would have to be an exposed podocyte protein, which led us to test for autoantibodies against nephrin, an essential component of the filtration barrier. Indeed, we identified anti-nephrin autoantibodies in a subset of adults and children with MCD both circulating in their blood and bound to nephrin in their kidney biopsy. This is a huge breakthrough in our understanding of this condition, as we can now define a subset of patients as suffering from an autoimmune disease. What are you looking at in your NephCure-funded grant, “Autoimmunity in Primary or Recurrent Podocytopathies – Clinicopathologic correlation and mechanistic studies”? In this grant, we are now extending our studies to patients with primary FSGS and recurrent FSGS after transplant. Since the changes to the podocytes in MCD and primary and recurrent FSGS are virtually indistinguishable, we hypothesized that a subset of patients with primary and recurrent FSGS also have circulating antibodies that target nephrin. Our preliminary findings demonstrate that this is indeed the case in a subset of patients from our own institution. Now we need to expand our studies to more samples. Can you tell us about treatment-resistant MCD versus steroid-sensitive MCD? The vast majority of MCD is steroid-sensitive, meaning that these patients will respond well to steroids. A small subset of these patients, however, will remain steroid-dependent, meaning they cannot come off this therapy or their Nephrotic Syndrome will relapse. This is not a good thing because 1) steroids have harsh side effects, especially during long-term use, and 2) often additional immunosuppressive agents are given that may add even worse side effects. Treatment-resistant patients never fully respond to steroids, and often receive a long list of unsuccessful immunosuppressive therapies, and despite all efforts may continue to spill protein and experience ongoing podocyte damage. Why do you think so many people are diagnosed with MCD to later be re-diagnosed with FSGS? Two possible reasons: 1) Sampling: With exception of the scarring, both MCD and Primary FSGS are indistinguishable on biopsy. Since we only look at a tiny sample of biopsied kidney tissue, and early FSGS can be very focal, we may miss scarring already present in your kidney. 2) Disease progression: A disease does not start out with scarring. Being diagnosed with MCD in your first and with FSGS in a later biopsy does not mean you were re-diagnosed; it just means that the initial injury was severe enough to have led to scarring during the course of the disease. Based on my experience, I believe that MCD and Primary FSGS are related and can represent expressions of the same initial disease process. This means that understanding one could enhance our understanding of the other. Why do you think there have not been trials for Minimal Change Disease patients until now? MCD has been generally perceived as a rather “benign” condition. Many patients respond well to a limited course of steroids, and complications remain rare, so there was no perceived acuity to start trials. We also haven’t had sufficient molecular understanding of this disease to provide a rationale for trying new or different treatments. Our studies may change how clinicians view this disease and may initiate such trials. Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on advancing kidney precision medicine. Its mission is to deliver disease-modifying precision medicine that brings hope and renewed quality of life to people living with kidney diseases. Goldfinch Bio’s TRACTION-2 clinical research trial is evaluating an investigational precision medicine, GFB-887, for the potential treatment of treatment-resistant Minimal Change Disease, FSGS, and Diabetic Nephropathy. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities and find the right trial for you, visit KidneyHealthGateway.com. This article was developed in partnership with Goldfinch Bio, Inc.
Everything You Need to Know About Travere’s New Clinical Trial for Children, EPPIK September 2, 2021 by Kylie Winkler Travere Therapeutics has recently launched their Phase 2 pediatric clinical trial, EPPIK (Evaluating Problematic Proteinuria in Kids) which is aimed at helping children with FSGS, MCD, IgAN and other rare kidney diseases. We know that with a clinical study specifically designed for kids, parents and caregivers may have some questions. We asked Travere to answer some of these questions and have complied an FAQ list for our community around EPPIK. What is the EPPIK study? EPPIK (Evaluating Problematic Proteinuria in Kids) is a Phase 2 study of children 1-17 years old with focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), IgA nephropathy (IgAN), also known as Berger’s disease, IgA vasculitis (IgAV), also known as Henoch-Schönlein purpura, or Alport syndrome (AS). These rare kidney diseases are associated with progression to end-stage kidney disease and currently have limited or no treatment options. This study aims to further understand if an investigational therapy, sparsentan, can help the kidneys filter protein and slow the decline of kidney function. The amount of protein found in the urine (called proteinuria) is seen as a marker of kidney function. Lowering proteinuria levels is associated with better kidney health outcomes. What is sparsentan? Sparsentan is a novel Dual Endothelin Angiotensin Receptor Antagonist (DEARA) – this means it is a dual acting, highly selective antagonist of both the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). In the EPPIK Study, sparsentan is taken once a day orally. What is a Phase 2 study? A Phase 2 study is a clinical trial that helps researchers determine how safe, tolerable and effective an investigational drug may be in specific patient populations, such as those noted above. While the EPPIK study is a Phase 2 study, sparsentan has been studied in another Phase 2 study as well as two Phase 3 studies involving approximately 880 patients (some of whom were children). The purpose of the EPPIK study is to gain further experience on the use of sparsentan in children. How long does this study take? Patients who meet all eligibility criteria and are enrolled in the study may expect to participate in the study for about 2 years and 3 months. Will you be enrolling children with congenital Nephrotic Syndrome or infantile Nephrotic Syndrome? Yes, children ages 1-17 with one of those syndromes who meet the study criteria will be able to participate in the EPPIK Study. The specific disorders we are looking for are noted above in the answer to question 1. How many children and young people are you looking to enroll in this study? We will be looking to enroll approximately 57 children in the EPPIK study. How many study sites are there available? Where are these sites located? Sites will be in the United States, UK, and various countries in the EU (France, Germany, Italy, Spain, Netherlands, Poland, Sweden). The final list of sites has not been fully determined yet. Because this study is for children, how is it different than a study for adults? The main difference is how much blood is drawn from the children to do all the tests necessary to evaluate sparsentan in the study. Patient safety is always our number one priority, and, with children, we won’t sample as much blood as we would from an adult. We narrowed the number of tests and worked with the central lab to be able to get results from a smaller sample size. We also must ensure that we adequately inform both the child who might participate in the study and the child’s parents/guardians about all the requirements the study has as well as any risks that may be associated with participating in the study. We do this by developing different study assents or ways to communicate what the study entails so that participating families understand the purpose of the study and what is involved. Are you asking kids about the taste, smell, etc. of the study drug? Yes, we will be asking the children to answer simple questions on how the drug tastes, smells, feels, etc. How do you determine which participants will be required to have pregnancy testing or take birth control? We realize this is a delicate subject for some children and their parents, but it is very important that any female child who has had her first menstruation period take birth control according to the protocol. This class of drugs is known to potentially cause harm to a developing fetus, and therefore we do not want any female participant who is able to have a child to become pregnant. Is there flexibility where lab samples can be collected for my child? Is there an option for home care visits to collect these samples? Yes, there is some flexibility in where labs are taken. If the child can’t make it to the clinical site to have the labs drawn, which is always the preferred way, we have contracted with a company that will come to the patient’s home or other convenient location, to draw the labs and send them in for testing. Does my child have to stop the medications they’re on? What drugs can they remain on? That depends, if they are enrolled in this study, they will need to stop any drug that functions similarly to the investigational drug. That is, if they are on an ACE inhibitor or ARB, they will need to stop those drugs at least 2 weeks before they begin taking the experimental drug and will not be able to take them while they are participating in the study. They will be able to continue with certain blood pressure medications if not in the same drug class, as well as many other medications they may be on. The Investigator will have a complete list of drugs that are allowed and not allowed. What will happen to the results of this clinical study? After all the patients have been enrolled and have completed the study, the data will be analyzed to see if it was successful in reducing the amount of protein in the urine and if it slowed the decline in kidney function as measured by estimated glomerular filtration rate, or eGFR. How well the drug was tolerated, that is, the safety profile, will also be analyzed. Those results will, in all likelihood, be put into a document known as a manuscript and published in a scientific journal. Data will also be added to the study listing on the clinicaltrials.gov website at the end of the study. If my child is in another trial studying sparsentan, do they have to enroll in this new study? If so, will their study site change? No, if your child is participating in another trial with sparsentan they would not be eligible to join the EPPIK study. They would stay in the study they are currently in. For more information about EPPIK, contact medinfo@travere.com
Kidney-Friendly Recipe: Italian Herb Grilled Chicken with Grilled Veggies and Quinoa September 1, 2021 by Kylie Winkler As kids head back to school and the long summer nights slowly slip away, celebrate the end of the season with a delicious meal on the grill—perfect for Labor Day Weekend! This recipe comes from NephCure’s Chef Sachet, a patient parent whose 12-year-old son Aiden was diagnosed with FSGS in January 2017. Aiden’s kidney disease rapidly progressed—just five months after diagnosis, he ended up on dialysis and eventually went on to receive a kidney transplant. As a kidney disease caregiver, Chef Sachet says ‘it’s important to me to keep kidney-friendly recipes in mind!’ Yield: 4 people Prep time: 15 min Cook time: 25 minutes Sodium: 139mg per serving Ingredients: 4 6-oz chicken breasts (with tenders attached) IMPORTANT NOTE: Check the sodium content on the chicken breasts to make sure they are no higher than 75 mg of sodium per 4 ounce serving or no higher than 113 mg per 6 ounce package. 2 cups of quinoa (any kind works) 1 large zucchini or squash 1 lime 1 lemon ½ handful of fresh parsley, minced 2 sprigs of fresh oregano, minced 2 sprigs of fresh thyme, minced 2 heads of fresh garlic, minced ¼ cup of extra virgin olive oil (EVOO) Pepper (to taste) 4 cups of no-salt veggie stock 2 Tbsp. of unsalted butter Instructions: First, set your chicken breasts aside in a large bowl. Mince all herbs and toss in chicken with garlic, pepper, lemon juice, and a drizzle of EVOO. Coat chicken well and marinate for 15 minutes. Heat the vegetable stock in large pot on medium-high heat until simmering. Add quinoa. Lower heat, add 2 Tbsp. of butter and a sprig of thyme. Let cook until all of the liquid is absorbed. Once most liquid is gone, turn off the heat, and cover the quinoa until serving time. Once the quinoa is cooking, cut vegetables into thick slices. Add pepper, EVOO, and a squeeze of lime, and then place them on the grill. Remove chicken from the fridge and start to grill it: 7-10 minutes on each side until the internal temperature reaches 165 degrees Fahrenheit. Chef’s Tips: Do not flip your chicken constantly when grilling. It will take a long time to cook and will dry out. Plus, you won’t get those awesome grill marks! Raw veggies can take a while on the grill. Season and lubricate them with oil ahead of time, but don’t add too much oil. It will just burn off and char the veggies. When using store-bought stock, always use low-sodium or no-salt varieties.
Encouraging interim results from Travere’s PROTECT trial for IgAN patients August 20, 2021 by Kylie Winkler Interim results have been announced from Travere Therapeutic’s PROTECT study, and although preliminary, the data look promising for IgA Nephropathy (IgAN) patients. Travere’s ongoing Phase 3 trial is studying the drug sparsentan in IgAN patients. On average, patients who received sparsentan experienced a nearly 50% reduction of proteinuria after 36 weeks. That’s roughly three times more than the active control. Active protein-spilling (proteinuria) leads to kidney damage. The priority for every patient should be to stop or reduce protein in their urine. To date, sparsentan has been generally well tolerated by those in the study. Unlike steroids and other commonly prescribed off-label drugs for progressive kidney diseases like IgAN, sparsentan is not an immunosuppressant. This news comes just a few months after the interim data from Travere’s Phase 3 DUPLEX study evaluating sparsentan in FSGS, also achieved its interim proteinuria endpoint. The PROTECT and DUPLEX studies are some of the largest studies to date for IgAN and FSGS patients, respectively, to reach this level of trial data. It’s possible that these results, could ultimately lead to a potential approval of sparsentan. To read more about Travere’s interim results from the PROTECT Study in IgAN, click here. Want to learn more about this research and what it could mean for patients with IgAN and other progressive, protein-spilling kidney diseases? Join NephCure’s Nurse Kristen on Facebook Live on August 25th where she discusses this news in further detail — breaking down the study results, who could be impacted by this research, and what the timeline could be to see new treatments for IgAN, FSGS, and other rare, protein-spilling kidney diseases.
The Liposorber Through the Eyes of Nephrologists July 1, 2021 by Kylie Winkler Kaneka’s LIPOSORBER® LA-15 System is a blood processing machine used outside of the body. This LDL apheresis device removes certain lipoproteins (cholesterol) from the blood. It’s used for pediatric and adult patients diagnosed with primary Focal Segmental Glomerulosclerosis (FSGS) either before transplant or who experienced FSGS recurrence after transplant. Dr. Joshua Zaritsky, MD, PhD Several doctors across the country have been treating patients with the LIPOSORBER®, including Dr. Joshua Zaritsky, a pediatric nephrologist, and Dr. Vasil Peev, an adult nephrologist. We asked both doctors to share their experience with the LIPOSORBER® system and recommendations for patients considering this treatment option. What is your experience with LDL apheresis, and what kind of patients do you treat? ZARITSKY: I’ve had experience with LDL apheresis over the last six years. There are two classes of patients I treat. One is patients who have had a renal transplant and have had recurrence — they’re probably the most common patients I treat. The other group of patients are those who have FSGS or another sort of steroid-resistant nephrotic syndrome. Those patients haven’t yet undergone transplantation. PEEV: I’ve treated two patients so far with LDL apheresis who have had recurrent FSGS and are post-transplant. What has the patient’s response been to the treatment? ZARITSKY: I’ve had very good luck with those patients who have had recurrence after transplant. We actually published that data; we had seven back-to-back cases that were 100% successful. Since then, we’ve had some failures to treatment, but we’ve had very good luck post-transplant. In the pre-transplant area, we see response rates anywhere from 30-50%. But that being said, we’ve had some incredible responses. Other centers were about to take out both patients’ kidneys, and we’ve been able to rescue them and put them into remission. Dr. Vasil Peev, MD PEEV: Both patients have had an outstanding response to LDL apheresis. One of them had recurrenceafter receiving a living-donor kidney from his mother. He’s now almost three years post-transplant with very stable renal function and no proteinuria. I just saw this patient recently and he’s doing fantastic! His kidney function remains somewhat impaired as his LDL apheresis treatment was initially delayed leading to some progression of CKD from recurrent FSGS after his transplant. When treating him after transplant, I initially gave him what is considered to be standard of care, i.e. plasmapheresis (and various other medications). He unfortunately failed these despite very aggressive and numerous efforts to control his proteinuria. The LDL apheresis was basically a Hail Mary treatment that really saved his kidney and kept him off dialysis. Are patients comfortable during the treatment? ZARITSKY: What I’ve noticed as a doctor is that this is very well-tolerated treatment. There’s not a lot of side effects. For the most part in my clinical experience, there hasn’t been a lot of side effects. PEEV: From what I’ve heard patients say, it’s very comfortable and much smoother than dialysis treatment. The blood flows are much lower (50-100 CCs per hour). The treatments last for few hours and are very well tolerated. Patients don’t feel drained, which is something that’s frequently described with dialysis. What is your recommendation for other nephrologists? ZARITSKY: I think it’s important to reach out to other physicians who have some experience using the equipment, because we can also put our nurses in touch with their nurses who have experience. There’s some tricks and tips that we’re willing to share and, for the most part, it’s a nice community of doctors who are always willing to help out. PEEV: I think that it’s definitely worth a try. Again, in the field of nephrology, unfortunately, we still are working with a handful of very old, adopted drugs from other medical specialties — mainly from oncology. I would convey the message to other nephrologists that the complication rates with this device are zero to none. Although it may require initially some efforts by the nephrologists to get patients treated with the LIPOSORBER® device (in terms of getting access to the device), these efforts will likely be eventually rewarded. In general, I think we should not spend much time with the other treatments if we see that they are not working or failing. Doctors should consider embarking on this treatment modality rather sooner, as sooner they expose their patients on this treatment (before more significant damage is done to the kidney), more likely the patients will have a good response. How early do you suggest starting the treatment? ZARITSKY: FSGS is one of these diseases that can aggressively decrease kidney function, and once that kidney function is gone, there’s no getting it back. The LIPOSORBER® is really one of these treatments that after patients have tried other treatment modalities, you want to treat them as soon as possible. PEEV: Ideally within the first 8-12 weeks after presentation. If you see the patients not responding to first-line therapies in 8-10 weeks following the initial presentation with recurrent FSGS, I would recommend initiating this treatment as soon as possible. When left untreated this condition is frequently associated with allograft loss, despite the best efforts of the transplant team. For more information about Kaneka’s LIPOSORBER® Trial, click here and here.
NephCure Kidney International Elects Longtime Volunteer Michael Levine as Board of Directors President June 15, 2021 by Kylie Winkler Levine succeeds NephCure co-founder Irving Smokler in the position Michael Levine, NephCure Board President, at Countdown to a Cure 2019 KING OF PRUSSIA, Pa. (June 15, 2021) — NephCure Kidney International (NephCure) announced today the election of longtime volunteer and board member Michael Levine as the new Board of Directors President. Levine, who joined NephCure’s Board of Directors in 2009, succeeds Irving Smokler, NephCure’s co-founder who has been with the organization since its inception in 2000. NephCure is one of the only organizations dedicated to finding better treatments and a cure for Nephrotic Syndrome and other rare, protein-spilling kidney diseases. “I’m beyond proud of all we have accomplished over the past 21 years,” said Smokler. “We’ve gone from knowing relatively nothing about Nephrotic Syndrome and FSGS to now a much better understanding of genetic causes and pathways, and we’re likely only a couple years away from having our first approved therapies. Some of this is due to the $40 million we’ve raised for research. It’s been a pleasure to be a part of this organization’s creation and success. I am confident in our future and in Michael’s leadership and passion for NephCure’s mission.” Levine has a special connection to NephCure — his son, Matthew, was diagnosed with focal segmental glomerulosclerosis (FSGS) in 2006, when he was only 2 years old. Shortly after Matthew’s diagnosis, Levine and his wife, Dana, joined NephCure in the pursuit of support and information. Before long, Levine became a dedicated volunteer, later advancing to the Board of Directors’ Executive Committee, which he’s served on since 2009. Levine takes a leading role in planning and running both Countdown to a Cure New York, NephCure’s largest annual fundraising event, and the yearly All In for a Cure golf tournament and banquet. These events have helped raise millions of dollars to support NephCure’s mission of saving kidneys and saving lives. Michael Levine with son, Matthew Levine Professionally, Levine has served as the president of L&L Painting for more than 14 years. The New York native’s deep passion for charity and helping others has also led him to join the Board of The Curetivity Foundation and actively contribute to various other charitable organizations throughout his life. “My mission has always been to help find a cure for all the warriors battling FSGS, Nephrotic Syndrome, and other chronic kidney diseases,” Levine said. “They are waiting for us to deliver a cure. We will not fail them under any circumstances — failure is not an option.” About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. 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 nearly 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. ###
Regulatory Update on Travere’s DUPLEX Study: Continuing on Accelerated Track June 4, 2021 by Kylie Winkler We know that many of you may have questions on the recent regulatory update from Travere Therapeutics on their sparsentan program for FSGS. We’re posting this to help you understand what it means: Travere reported that despite the DUPLEX Study achieving its interim endpoint, the FDA indicated in recent interactions that the data from the DUPLEX Study are not yet adequate enough to support accelerated approval Travere noted that FDA has acknowledged the high unmet need and the limited treatment options for people living with FSGS For anyone participating in or following the DUPLEX Study, there are no changes to report at this time. The study will continue as planned to the final endpoint after 108 weeks of treatment Travere is continuing to engage with regulators about generating additional data from DUPLEX in the first half of next year with the gold of submitting for accelerated approval in 2022 This means that sparsentan could potentially gain accelerated approval in the U.S. in 2023 Patient retention in the study is critical to providing the FDA with most robust data package possible to support a potential approval at the appropriate time Travere remains confident in the potential for sparsentan to become a new treatment standard for FSGS, if approved Travere also recently announced that their Phase 3 PROTECT Study in IgAN has completed enrollment and interim data from the trial are expected in August of this year The interim data could also lead to an accelerated approval submission for IgAN To read Travere Therapeutics’ original press release, click here. Still want to learn more about how a drug gets approved by the FDA? Here is an FAQ list to help you find the information you’re seeking. How does the FDA determine which study drugs are eligible for accelerated approval and which follow the normal regulatory timeline? The purpose of the FDA’s accelerated approval program is to get important new drugs to patients in need earlier. Generally speaking, the drug must have an impact on factors like survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more seriousone. The drug must fulfill an unmet medical need, which the FDA defines as providing a therapy where none exists or providing a therapy which may be potentially better than an available therapy. How does this news impact my participation in the trial? This news doesn’t change anything about your participation in this study. In fact, it’s very important that you remain in the study. Your continued participation is the only way Travere’s sparsenten will retain the potential to be granted accelerated approval. Please continue to do everything you are currently doing and comply with all study protocol/requirements. Be sure to talk to your study doctor if you have questions. The FDA’s response to Travere’s request for accelerated approval was, ‘the available data from the interim assessment of the DUPLEX Study would not be adequate to support an accelerated approval at this time.’ Will they be able to submit for accelerated approval at a later time? Although Travere was not granted accelerated approval at this time, they may still be able to submit for accelerated approval once additional data accrue in the DUPLEX Study. Subject to further discussions this summer, those data may become available to FDA in the first half of 2022. What does this mean for Europe? Travere continues to work through the conditional marketing authorization process and anticipates meeting with European regulators this summer to discuss next steps. Subject to their upcoming discussions with EMA, Travere is still planning for a submission in Europe this year. They expect to provide an update in the coming months.
Do you have Focal Segmental Glomerulosclerosis (FSGS)? June 1, 2021 by Kylie Winkler Have you or your loved one been diagnosed with FSGS? We have resources for you. The rates of severe kidney disease are high in individuals who are African American, Hispanic black, Afro-Caribbean, or of African ancestry. This could be due to differences in the genetic makeup in the APOL1 gene found typically in individuals with recent African, Caribbean, or Latin American descent. These differences in the genetic makeup are associated with increased rates of hypertension-associated kidney failure, FSGS, HIV-associated kidney disease, and other forms of nondiabetic kidney disease. Fast facts about APOL1 FSGS: Black Americans account for 32% of all kidney failure in the US Black Americans are four times more likely to develop kidney failure than White Americans Approximately 4 in 10 Black Americans on dialysis have kidney failure caused by APOL1 gene changes Approximately 1 in 5 people with two copies of the APOL1 gene changes will develop kidney disease The high-risk APOL1 genotype is present in 75% of Black patients with FSGS Below are resources we’ve complied that might be useful for you: This informational flyer on APOL1 FSGS breaks down the basics and helps you better understand this disease. To download the full informational sheet, click here. In June 2020, we hosted a NephCure U session specifically on APOL1-Associated FSGS. Dr. Jeffrey Kopp from the NIDDK lead, “Kidney Disease in Patients of African Descent: APOL1-Associated Disease” and discussed more on the diagnosis, treatment options, and clinical trials. Listen in on the hour-long educational webinar below. NephCure co-hosted a GlomCon Clinical Trial Conference Series session on Advances in APOL1 Therapeutics on February 14, 2021, featuring Dr. Ogo Egbuna, Dr. Opeyemi Olabisi, and Dr. David J. Friedman. Click here to watch the recording of this session. In addition to these resources, there are also clinical trials available for APOL1-Associated FSGS patients. Clinical trials look at the safety and effectiveness of potential new treatments. The main goals of clinical trials are to find new ways to prevent, detect (find) or treat diseases or health conditions, and to make sure potential new treatments or therapies work well and are safe for people. Check out some pre-screener questions below, provided by Vertex, to see if their clinical trial could be a fit for you or your loved one. Eligible participants must meet the following criteria: Be male or female adults between the ages of 18 and 65 (inclusive) Female participants must not be pregnant or breast-feeding Be of African, Caribbean or Latin American descent Have had a kidney biopsy which has found focal segmental glomerulosclerosis (FSGS) Have not had a diagnosis of kidney disease other than FSGS Be willing to complete the investigational apolipoprotein L1 (APOL1) gene test Be willing and able to follow the study instructions To learn more about Vertex’s clinical trial, click here. If you meet the preliminary criteria listed above, find the location closest to you and click the “I’m Interested” button to get in touch with an investigator for additional evaluation of eligibility. You can find a full list of clinical trials for all protein-spilling kidney diseases on KidneyHealthGateway.com.
Giving Tuesday, Nov. 30th May 24, 2021 by Kylie Winkler Join the global giving movement — help us raise critical funds this Giving Tuesday.