Vadadustat

Akebia Therapeutics said Tuesday it's abandoning plans to reach more chronic kidney disease (CKD) patients with its anaemia treatment, prompting the company's shares to plummet more than 20% during after-hours trading.While the FDA approved Vafseo (vadadustat) last year in adults with CKD who have been receiving dialysis for at least three months, Akebia was shooting for a broader label that included patients not on dialysis — an indication the US regulator had previously rejected back in 2022. At the time, the agency had flagged safety and efficacy concerns, noting that Vafseo failed to demonstrate non-inferiority with regard to major adverse cardiovascular events (MACE) versus Amgen's Aranesp (darbepoetin alfa) in CKD patients not dependent on dialysis.Still, Akebia sought to expand the once-daily oral HIF-PH inhibitor's label; in its second-quarter financial report, the company said it was planning to launch the Phase III VALOR trial of Vafseo to treat anaemia in late-stage CKD patients not on dialysis by year-end.However, Akebia said Tuesday that during a Type C meeting with the FDA, they could not "come to alignment on a path forward for the design of the VALOR clinical trial." Because VALOR would require more patient enrollment than Akebia had proposed, and thus more time and money, the company will not launch the trial and is no longer pursuing an approval for Vafseo in non-dialysis dependent (NDD)-CKD patients."We are disappointed in the outcome of the meeting,” said CEO John P. Butler. "We believe our decision not to pursue a broad NDD-CKD label, based on our discussion with the FDA, is in the best interests of our shareholders."

Presentations Detail Ongoing Vafseo® (Vadadustat) Real-World Studies and Explore Insights Regarding Clinical Benefits and Dosing of VafseoCAMBRIDGE, Mass., Oct. 20, 2025 (GLOBE NEWSWIRE) -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced that it will present data at the American Society of Nephrology Kidney Week 2025 (ASN Kidney Week), which will take place in Houston, TX from November 5-9, 2025. ASN Kidney Week attendees can also visit Akebia at Booth #903 in the Exhibit Hall. Dr. Glenn M. Chertow of Stanford University will present the results of a win odds analysis of all-cause mortality and hospitalization from the INNO2VATE trials, part of the global Phase 3 clinical development programs for vadadustat. In the U.S., Vafseo® (vadadustat) is approved for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Additionally, four posters will present data to inform dosing of vadadustat as well as information on ongoing real-world evidence studies, including the VOICE and VOCAL trials. “We remain actively engaged with the nephrology community as we conduct multiple clinical real-world evidence studies and identify potential additional clinical benefits of Vafseo,” said Steven Burke, MD, Senior Vice President and Chief Research & Development Officer at Akebia. “Generating, evaluating and sharing data are critical to ensuring healthcare providers have the information they need to make informed decisions about treating patients with Vafseo and to further the medical and scientific communities’ understanding about this new, innovative medicine.” Oral presentation details: Win-Odds Analysis of Deaths and Hospitalization in Patients taking Vadadustat or Darbepoetin Alfa for CKD-Related Anemia Undergoing DialysisPresenter: Glenn M. Chertow, MDSession Title: Hemodialysis: Novel Interventions Session Date/Time: November 6, 2025; 4:30 PM to 6:00 PMSession Room: Room 351D (Convention Center) Akebia-supported posters to be presented at ASN Kidney Week 2025 in the Exhibit Hall: Model-Based VAFSEO Starting Dose Recommendations in Dialysis-Dependent CKD Patients Poster #: TH-PO902November 6, 2025; 10:00 AM-12:00 PM CDT Vadadustat Outcomes In-Center Experience (VOICE) Study: Pragmatic Randomized Controlled Trial to Test the Safety of Three Times Weekly Vadadustat Poster #: FR-PO0204November 7, 2025; 10:00 AM-12:00 PM CDT Consistency of the Estimate of Responsiveness of Vadadustat vs. Darbepoetin Poster #: FR-PO0202 November 7, 2025; 10:00 AM-12:00 PM CDT Three Times Weekly In-Center Vadadustat vs. Standard of Care Erythropoiesis-Stimulating Agent (ESA) for Treatment of CKD-Related Anemia in Patients Undergoing Dialysis (VOCAL)Poster #: INFO10-FRNovember 7, 2025; 10:00 AM-12:00 PM CDT About Akebia TherapeuticsAkebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at www.akebia.com, which does not form a part of this release. About Vafseo® (vadadustat) tablets Vafseo® (vadadustat) tablets is a once-daily oral hypoxia-inducible factor prolyl hydroxylase inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin, increasing hemoglobin and red blood cell production to manage anemia. Vafseo is approved for use in 37 countries. INDICATION VAFSEO is indicated for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Limitations of Use VAFSEO has not been shown to improve quality of life, fatigue, or patient well-being.VAFSEO is not indicated for use: As a substitute for red blood cell transfusions in patients who require immediate correction of anemia.In patients with anemia due to CKD not on dialysis. IMPORTANT SAFETY INFORMATION about VAFSEO (vadadustat) tablets WARNING: INCREASED RISK OF DEATH, MYOCARDIAL INFARCTION, STROKE, VENOUS THROMBOEMBOLISM, and THROMBOSIS OF VASCULAR ACCESS. VAFSEO increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE). Targeting a hemoglobin level greater than 11 g/dL is expected to further increase the risk of death and arterial and venous thrombotic events, as occurs with erythropoietin stimulating agents (ESAs), which also increase erythropoietin levels. No trial has identified a hemoglobin target level, dose of VAFSEO, or dosing strategy that does not increase these risks. Use the lowest dose of VAFSEO sufficient to reduce the need for red blood cell transfusions. CONTRAINDICATIONS Known hypersensitivity to VAFSEO or any of its componentsUncontrolled hypertension WARNINGS AND PRECAUTIONS Increased Risk of Death, Myocardial Infarction (MI), Stroke, Venous Thromboembolism, and Thrombosis of Vascular Access A rise in hemoglobin (Hb) levels greater than 1 g/dL over 2 weeks can increase these risks. Avoid in patients with a history of MI, cerebrovascular event, or acute coronary syndrome within the 3 months prior to starting VAFSEO. Targeting a Hb level of greater than 11 g/dL is expected to further increase the risk of death and arterial and venous thrombotic events. Use the lowest effective dose to reduce the need for red blood cell (RBC) transfusions. Adhere to dosing and Hb monitoring recommendations to avoid excessive erythropoiesis. Hepatotoxicity Hepatocellular injury attributed to VAFSEO was reported in less than 1% of patients, including one severe case with jaundice. Elevated serum ALT, AST, and bilirubin levels were observed in 1.8%, 1.8%, and 0.3% of CKD patients treated with VAFSEO, respectively. Measure ALT, AST, and bilirubin before treatment and monthly for the first 6 months, then as clinically indicated. Discontinue VAFSEO if ALT or AST is persistently elevated or accompanied by elevated bilirubin. Not recommended in patients with cirrhosis or active, acute liver disease. Hypertension Worsening of hypertension was reported in 14% of VAFSEO and 17% of darbepoetin alfa patients. Serious worsening of hypertension was reported in 2.7% of VAFSEO and 3% of darbepoetin alfa patients. Cases of hypertensive crisis, including hypertensive encephalopathy and seizures, have also been reported in patients receiving VAFSEO. Monitor blood pressure. Adjust anti-hypertensive therapy as needed. Seizures Seizures occurred in 1.6% of VAFSEO and 1.6% of darbepoetin alfa patients. Monitor for new-onset seizures, premonitory symptoms, or change in seizure frequency. Gastrointestinal (GI) Erosion Gastric or esophageal erosions occurred in 6.4% of VAFSEO and 5.3% of darbepoetin alfa patients. Serious GI erosions, including GI bleeding and the need for RBC transfusions, were reported in 3.4% of VAFSEO and 3.3% of darbepoetin alfa patients. Consider this risk in patients at increased risk of GI erosion. Advise patients about signs of erosions and GI bleeding and urge them to seek prompt medical care if present. Serious Adverse Reactions in Patients with Anemia Due to CKD and Not on Dialysis The safety of VAFSEO has not been established for the treatment of anemia due to CKD in adults not on dialysis and its use is not recommended in this setting. In large clinical trials in adults with anemia of CKD who were not on dialysis, an increased risk of mortality, stroke, MI, serious acute kidney injury, serious hepatic injury, and serious GI erosions was observed in patients treated with VAFSEO compared to darbepoetin alfa. Malignancy VAFSEO has not been studied and is not recommended in patients with active malignancies. Malignancies were observed in 2.2% of VAFSEO and 3.0% of darbepoetin alfa patients. No evidence of increased carcinogenicity was observed in animal studies. ADVERSE REACTIONS The most common adverse reactions (occurring at ≥ 10%) were hypertension and diarrhea. DRUG INTERACTIONS Iron supplements and iron-containing phosphate binders: Administer VAFSEO at least 1 hour before products containing iron.Non-iron-containing phosphate binders: Administer VAFSEO at least 1 hour before or 2 hours after non-iron-containing phosphate binders.BCRP substrates: Monitor for signs of substrate adverse reactions and consider dose reduction.Statins: Monitor for statin-related adverse reactions. Limit the daily dose of simvastatin to 20 mg and rosuvastatin to 5 mg. USE IN SPECIFIC POPULATIONS Pregnancy: May cause fetal harm.Lactation: Breastfeeding not recommended until two days after the final dose.Hepatic Impairment: Not recommended in patients with cirrhosis or active, acute liver disease. Please note that this information is not comprehensive. Please click here for the Full Prescribing Information, including BOXED WARNING and Medication Guide. Forward-Looking Statements Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia’s plans and expectations with respect to its ability to identify potential additional clinical benefits of Vafseo; and Akebia’s beliefs that generating, evaluating and sharing data are critical to ensuring healthcare providers have the information they need to make informed decisions about treating patients with Vafseo and to further the medical and scientific communities’ understanding about this new, innovative medicine. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: the potential therapeutic benefits, safety profile, and effectiveness of Vafseo; the results of preclinical and clinical research; decisions made by health authorities, such as the FDA, with respect to regulatory filings and other interactions; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Vafseo, including estimates regarding the potential market opportunity; the competitive landscape for Vafseo, including generic entrants and the timing thereof; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to achieve and maintain profitability and to maintain operating expenses consistent with its operating plan; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; early termination of any of Akebia's collaborations; and changes in the geopolitical environment and uncertainty surrounding U.S. trade policy on tariffs. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Akebia Therapeutics® and Vafseo® are registered trademarks of Akebia Therapeutics, Inc. Akebia Therapeutics ContactMercedes Carrascomcarrasco@akebia.com