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iStock , Lara_Uhryn Disc Medicine’s bitopertin—rejected by the FDA in February—significantly reduced levels of a key disease biomarker and significantly increased the time patients with erythropoietic protoporphyria were able to spend in the sun and artificial light. Disc Medicine’s bitopertin helped patients with a rare disorder that causes painful sensitivity to the sun spend more time in the light during a pivotal trial, setting the drug up for another attempt at an FDA approval after an accelerated application was rejected last year . When the results were unblinded “a few days ago,” Disc CEO John Quisel was “overjoyed, as was our whole team,” he told BioSpace in an interview. “Just thrilled to be able to deliver a set of clean results in a set of endpoints that can be tricky.” The results, announced on Monday, revealed that the Phase 3 APOLLO trial hit all primary and secondary endpoints. Prior to the results, analysts from BMO Capital Markets were optimistic that the readout could clear the way for a regulatory approval in erythropoietic protoporphyria (EPP). Senior Biopharma Analyst Evan Seigerman told BioSpace that the firm believed there was a high probability that APOLLO would deliver. Seigerman said bitopertin’s established ability to lower PPIX and the APOLLO trial’s sunlight-exposure endpoint could give Disc a clear path back to regulators. Pivotal rare disease trials are designed to show clear clinical benefit, Seigerman explained. “If that trial hits, then there’s a real path forward for approval.” The regulatory road for bitopertin to this point has been tricky. In its complete response letter (CRL), revealed in a February 13 SEC filing by Disc, the FDA questioned the biotech’s use of a surrogate endpoint—percent change in whole-blood metal-free protoporphyrin IX (PPIX), the toxic metabolite that drives the disease—as a reasonable predictor of clinical benefit in EPP. “The percent change in PPIX was relatively modest,” the agency wrote in the CRL, noting that it is unknown whether such magnitude of biomarker effect “is reasonably likely to predict clinical benefit.” Bitopertin was one of the first recipients of the Commissioner’s National Priority Voucher in October 2025, granted to products that align with certain national priorities, such as meeting a large unmet medical need or boosting domestic manufacturing. A CNPV entitles the product to a drastically shortened review period of one to two months, down from the typical 10–12 months—but it does not guarantee approval. In June, Disc announced it had reached an agreement with the FDA whereby the regulator would allow the use of the ongoing APOLLO trial, if successful, to support another regulatory filing for bitopertin. With the new data in hand, Disc plans to resubmit its application—this time for traditional approval of bitopertin—to the FDA by end of the year. Complete response letters FDA Rejects Disc’s Rare Blood Disease Drug Amid Reports of Prasad’s Skepticism The FDA in October 2025 granted bitopertin its Commissioner’s National Priority Voucher but after a shortened review time has decided the data did not support regulatory approval for treating erythropoietic protoporphyria. February 16, 2026 · 3 min read · Tristan Manalac Read more Bitopertin soars in Phase 3 The APOLLO trial, which enrolled 183 adults and adolescents with EPP, showed that bitopertin reduced PPIX by 41.4% compared to placebo (p-value < 0.0001) and increased time patients were able to spend in the light by 12.2 hours during month six (p-value = 0.0010), meeting the study’s two co-primary endpoints with what Quisel called a “high” degree of statistical significance. All secondary endpoints were also positive, Disc said, including a statistically significant reduction in phototoxic reactions—which Quisel described as “horrifically painful” attacks where the skin is damaged—and increased light tolerance. Exploratory measures of quality-of-life improvement were also statistically significant. As to the pain attacks, Quisel elaborated on the experience patients have relayed to Disc. “What patients tell us is it feels like they’re being burned or cooked from the inside, because this chemical, PPIX, is floating around in their blood, and when it’s activated by the light, it literally is burning them from the inside.” Many patients receive opioids, even though they don’t address the pain effectively, he added. Bitopertin was generally well-tolerated with no new safety signals observed in APOLLO, according to Disc’s press release. Dizziness was the most common treatment-emergent adverse event, with 50% of patients in the treatment group experiencing this side effect, but the cases were mild to moderate and generally transient, the company said. In addition, there were no study discontinuations in the bitopertin arm of the study, “which is remarkable for a six-month study,” Quisel said. Collectively, the results will support a fresh new drug application for bitopertin. When that filing reaches the FDA, it will face a regulator that looks quite different than it did in February. Former Commissioner Marty Makary, Center for Biologics Evaluation and Research (CBER) director Vinay Prasad and Center for Drug Evaluation and Research (CDER) Acting Director Tracy Beth Høeg have all departed the agency. Prasad reportedly intervened in the initial review of bitopertin, expressing his skepticism about the drug’s efficacy. If Disc’s drug is cleared for the market, it will join Clinuvel Pharmaceuticals’ Scenesse, a subcutaneous implant that is the only treatment approved for EPP and to prevent phototoxic reactions. Disc began development of bitopertin in 2021 when the company gained global rights to the asset from Roche. At the time, Disc paid an undisclosed upfront fee for the candidate and offered Roche more than $200 million in potential milestone payments. Still to come Beyond bitopertin, BMO is bullish on Disc because of the company’s two other clinical assets. One of those is a monoclonal antibody called DISC-3405, designed to increase the production of hepcidin—a key regulator of iron entry into circulation—to therapeutically reduce iron levels. The drug, licensed from Mabwell Therapeutics in 2023, is being studied in a Phase 2 trial for polycythemia vera (PV) and a Phase 1b study for sickle cell disease. In early September, Disc said that DISC-3405 increased hepcidin and lowered serum iron in patients with PV, translating to reduced blood draws and symptom burden, according to a poster presentation at the 2026 Society of Hematologic Oncology meeting. Cancer Disc sets up polycythemia vera challenge but Silence maintains lead The polycythemia vera field is heating up, with Disc Medicine and Silence Therapeutics each advancing next-generation therapies, playing catch-up to Takeda and Protagonist Therapeutics, who won the FDA’s approval last month for Mimrylo. September 11, 2026 · 2 min read · Tristan Manalac Read more Disc’s other asset is a monoclonal antibody called DISC-0974 picked up from AbbVie upon the biotech’s launch in 2019. Earlier this year, Disc shared that the asset reduced hepcidin and increased iron levels for patients with myelofibrosis (MF) and anemia in a Phase 2 trial called RALLY-MF . Additional data from RALLY-MF are slated to read out sometime this quarter and are expected to “further clarify the asset’s differentiated pro myelofibrosis anemia, with end of Phase 2 discussions with FDA providing more details on future Ph 3 trial design,” according to a September report from BMO. As for bitopertin, Disc said in its press release that an FDA decision is expected by mid-2027. Asked if, given the previous allocation of a CNPV, the agency might move faster than the six months typical for a resubmission, Quisel said it’s possible. “They have a lot of priorities, so we will certainly ask, and we’ll see what we get.”

A diversified late-stage pipeline encompassing endothelin receptor antagonists, aldosterone synthase inhibitors such as lorundrostat, APOL1 inhibitors including MZE829, and cell therapy approaches such as rilparencel is poised to intensify competition, with each modality addressing mechanisms that complement the established SGLT2 inhibitor/MRA backbone. The CKD market expansion is further expected to be supported by rising diagnosis rates, earlier screening based on UACR and eGFR, and broader adoption of combination therapies. LAS VEGAS, Sept. 24, 2026 /PRNewswire/ -- Treatment of chronic kidney disease (CKD) focuses on slowing disease progression toward kidney failure, reducing cardiovascular risk, and addressing complications such as anemia and mineral bone disorders. SGLT2 inhibitors, non-steroidal mineralocorticoid receptor antagonists (MRAs), and emerging aldosterone synthase inhibitor combinations are increasingly replacing single-agent RAS blockade, driven by their improved renal and cardiovascular outcomes. Approved therapies in the CKD market include empagliflozin (JARDIANCE) (Boehringer Ingelheim and Eli Lilly), finerenone (KERENDIA) (Bayer), dapagliflozin (FARXIGA/FORXIGA) (AstraZeneca), among others. Find out more about what is the latest treatment for chronic kidney disease @ The pharmaceutical companies involved in the emerging pipeline include AstraZeneca (zibotentan/dapagliflozin), Boehringer Ingelheim (vicadrostat + empagliflozin), Mineralys Therapeutics (lorundrostat), ProKidney (rilparencel), Disc Medicine (DISC-0974), and others. Aparna Thakur, Project Manager of Forecasting & Analytics at DelveInsight, said that Rilparencel and MZE829 represent potential disease-modifying and precision-genetic opportunities, respectively, with the potential to achieve premium positioning in advanced and APOL1-driven CKD if pivotal data continue to be positive. Thakur further added that DISC-0974 could establish a new anemia-focused revenue segment within the broader CKD market, with limited direct competition from therapies primarily targeting SGLT2i- and MRA-focused mechanisms. Although SGLT2 inhibitors, non-steroidal MRAs, and RAS blockade have been approved, a significant proportion of CKD patients continue to progress to kidney failure, while the disease remains substantially underdiagnosed. This highlights the need for more effective therapies to slow or halt disease progression, alongside improved strategies for early detection and diagnosis. Reflecting the growing disease burden and significant unmet medical needs, the chronic kidney disease market across the seven major markets, comprising the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, was valued at approximately USD 5 billion in 2025. The market is projected to expand at a significant CAGR during the forecast period through 2036, driven by the increasing prevalence of CKD, rising awareness and diagnosis, advances in disease management, and the introduction of novel therapies targeting disease progression and associated complications. Below, we highlight 5 promising emerging therapies poised to reshape the future of the chronic kidney disease market. AstraZeneca's Zibotentan/Dapagliflozin Endothelin receptor antagonist/SGLT2 inhibitor Zibotentan/Dapagliflozin, developed by AstraZeneca, is an investigational oral combination therapy being studied in adults with CKD. The therapy combines zibotentan, an endothelin receptor antagonist, with dapagliflozin, an SGLT2 inhibitor, with the goal of improving kidney outcomes by targeting complementary mechanisms involved in CKD progression. Following encouraging Phase IIb ZENITH-CKD results, which demonstrated up to a 52.5% reduction in urine protein markers, enrollment in the Phase III ZENITH High Proteinuria trial was completed in late 2024, with topline results anticipated in 2027. Boehringer Ingelheim's Vicadrostat (BI 690517) + Empagliflozin Aldosterone synthase inhibitor/SGLT2 inhibitor Vicadrostat + empagliflozin, developed by Boehringer Ingelheim, is an investigational combination therapy being studied in adults with CKD. In a Phase II CKD trial conducted in 2023, the combination demonstrated promising results, with full data showing a significant reduction of up to 39.5% in excessive urinary protein levels (albuminuria) compared with placebo. The vicadrostat and empagliflozin combination may provide a new treatment option for people living with CKD. Discover more about the chronic kidney disease treatment market @ Chronic Kidney Disease Drugs Market ProKidney's Rilparencel Stabilizes eGFR decline ProKidney is developing rilparencel, an investigational cell therapy product designed to preserve kidney function by stabilizing estimated glomerular filtration rate (eGFR) in patients with advanced CKD. The company is developing rilparencel with the potential to delay or prevent the need for dialysis. Rilparencel is a percutaneous, minimally invasive injectable product prepared from the patient's own kidney cells. As rilparencel is prepared from the patient's own cells, immunosuppressive therapy to prevent rejection is not required. To date, clinical studies suggest that rilparencel treatment in patients with advanced CKD and diabetes may positively impact kidney function by stabilizing eGFR or attenuating the rate of eGFR decline. Mineralys Therapeutics' Lorundrostat Aldosterone synthase inhibitor Lorundrostat, developed by Mineralys Therapeutics, is an investigational oral aldosterone synthase inhibitor being studied in adults with CKD. The therapy selectively inhibits aldosterone production, targeting hormonal pathways implicated in the progression of CKD. In January 2026, Mineralys Therapeutics provided a corporate update outlining recent and anticipated clinical and regulatory milestones for Lorundrostat in CKD and related conditions. In November 2025, Mineralys Therapeutics reported data on Lorundrostat in CKD and hypertension at ASN Kidney Week 2025, organized by the American Society of Nephrology. In June 2025, Mineralys Therapeutics announced positive topline results from the Phase II EXPLORE-CKD trial evaluating Lorundrostat in patients with CKD and albuminuria. To know more about what new drug approvals have there been for chronic kidney disease treatment in the US recently, visit @ Chronic Kidney Disease Medications Disc Medicine's DISC-0974 DISC-0974 is an investigational anti-hemojuvelin (HJV) monoclonal antibody designed to suppress hepcidin production and enhance iron levels for the treatment of anemia of inflammation. DISC-0974 was in-licensed from AbbVie and is being evaluated in multiple clinical studies. Disc completed a Phase 1 clinical study in healthy volunteers and has advanced the program into development for multiple forms of anemia of inflammation. The company has initiated a Phase 1b/2 study in patients with anemia of myelofibrosis (MF) and a Phase 2 study in patients with anemia of inflammatory bowel disease (IBD). Download the report to understand the top chronic kidney disease drugs manufacturer @ Chronic Kidney Disease Drugs Supplier Source: Chronic Kidney Disease Market Report Chronic Kidney Disease Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key CKD companies, including Boehringer Ingelheim, Eli Lilly, Bayer, AstraZeneca, Mineralys Therapeutics, ProKidney, Maze Therapeutics, Regeneron Pharmaceuticals, Disc Medicine, and others. Related Reports Chronic Kidney Disease Clinical Trial Analysis Chronic Kidney Disease Pipeline Insight – 2026 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CKD companies, including Novo Nordisk, Prokidney, Kibow Pharma, XORTX Therapeutics, Lisata Therapeutics, Unicycive Therapeutics, Novo Nordisk, Bayer, AstraZeneca, Roche, MC2 Therapeutics, Allena Pharmaceuticals, Boehringer Ingelheim, SCOHIA PHARMA, Regeneron Pharmaceuticals, UnicoCell Biomed, OccuRx, Disc Medicine, Shandong Suncadia Medicine, Sarfez Pharmaceuticals, Rege Nephro, Orgenesis, Healx, Benevolent, and others. Anemia in Chronic Kidney Disease Market Anemia in Chronic Kidney Disease Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key anemia in chronic kidney disease companies, including Jiangsu HengRui Medicine, Shenyang Sunshine Pharmaceutical, Biocad, Xenetic Biosciences, Chiasma, Liminal BioSciences, Acceleron Pharma, Celgene Corporation, and others. Moderate and Severe Chronic Kidney Disease Market Moderate and Severe Chronic Kidney Disease Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key moderate and severe chronic kidney disease companies, including Kibow Pharma, Bayer, Roche, Caladrius Biosciences, UnicoCell Biomed, Pharmicell, Scohia Pharma, Unicycive Therapeutics, and others. End-Stage Renal Disease Market End-Stage Renal Disease Market Insight, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key ESRD companies, including Medtronic PLC, B. Braun SE, Asahi Kasei Medical Co. Ltd, Cantel Medical (STERIS), Nikkiso Co. Ltd, Nipro Corporation, Fresenius SE & Co. KGaA , and others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 SOURCE DelveInsight Business Research, LLP 21% more press release views with Request a Demo