Volixibat

The Cushing's disease pipeline possesses some drugs in mid and late-stage development to be approved in the near future. The emerging landscape holds a diverse range of therapeutic alternatives for treatment, including relacorilant, clofutriben, atumelnant, and others. The expected launch of these therapies shall further create a positive impact on the Cushing's disease market. LAS VEGAS, May 15, 2025 /PRNewswire/ -- DelveInsight's Cushing's Disease Market Insights report includes a comprehensive understanding of current treatment practices, Cushing's disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Cushing's Disease Market Report According to DelveInsight's analysis, the market size for Cushing's disease was found to be USD 1 billion in the 7MM in 2024. In 2024, among all the current therapies for Cushing's disease, the highest revenue was generated by Mifepristone (KORLYM and generics) in the US. Based on DelveInsight's assessment in 2024, the 7MM had approximately 37K diagnosed prevalent cases of Cushing's disease. These cases are expected to rise due to advancements in diagnostic capabilities and increased awareness about the disease during the forecast period (2025–2034). Leading Cushing's disease companies developing emerging therapies, such as Corcept Therapeutics, Sparrow Pharmaceuticals, Crinetics Pharmaceuticals, H. Lundbeck, Stero Therapeutics, and others, are developing novel Cushing's disease drugs that can be available in the Cushing's disease market in the coming years. The promising Cushing's disease therapies in the pipeline include DUPIXENT (dupilumab), Linerixibat (GSK2330672), NEMLUVIO/MITCHGA (nemolizumab), Volixibat, and others. In March 2025, Corcept Therapeutics announced that the FDA had assigned a PDUFA target action date of December 30, 2025, for relacorilant to treat patients with endogenous hypercortisolism. In February 2025, Crinetics Pharmaceuticals mentioned that the company anticipates beginning enrollment for Atumelnant (CRN04894) in Phase Ib/IIa trials for Cushing's disease in late 2025 or early 2026. In October 2024, Sparrow Pharmaceuticals announced that clofutriben had been granted Orphan Drug Designation (ODD) by the US FDA for the treatment of endogenous Cushing's syndrome. Discover which therapies are expected to grab the major Cushing's disease market share @ Cushing's Disease Market Report Cushing's Disease Overview Cushing's disease is a rare but serious endocrine disorder caused by a pituitary tumor that triggers excessive cortisol production. It presents with a variety of symptoms such as weight gain, fatigue, mood disturbances, high blood pressure, and impaired glucose tolerance. Cushing's syndrome is responsible for 80–85% of hypercortisolism cases, with 75–80% of those linked to ACTH-producing pituitary adenomas. Without treatment, the 5-year survival rate is only about 50%, emphasizing the urgent need for effective therapies. Diagnosing Cushing's disease is especially difficult because it is rare, shares symptoms with other disorders, and presents in various ways. Only 40–60% of patients have a detectable tumor on standard Magnetic Resonance Imaging (MRI), and the diagnosis is often delayed by an average of 7 years. Cushing's Disease Epidemiology Segmentation The Cushing's disease epidemiology section provides insights into the historical and current Cushing's disease patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Cushing's disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Cushing's Disease Total Diagnosed Prevalent Cases of Cushing's Disease Gender-Specific Diagnosed Prevalent Cases of Cushing's Disease Age-Specific Diagnosed Prevalent Cases of Cushing's Disease Cushing's Disease Treatment Market The primary treatment for Cushing's disease is usually transsphenoidal surgery to remove the pituitary tumor, which often leads to remission. However, some patients may experience a recurrence. When surgery is unsuccessful or not an option, medical therapies are used to control cortisol levels. Various medications are available for managing Cushing's disease, especially in cases where surgery is ineffective or not feasible. One such drug is KORLYM, developed by Corcept Therapeutics, approved for treating hyperglycemia in patients with Cushing's syndrome who also have Type 2 diabetes or glucose intolerance. In 2024, a generic version by Teva Pharmaceuticals entered the market, adding competition, and Corcept itself has released a generic version as well. Other approved options include S IGNIFOR and SIGNIFOR LAR, ISTURISA, and RECORLEV. In addition, off-label drugs like ketoconazole, metyrapone, cabergoline, mitotane, and etomidate are also used in the U.S., with some marketed in Europe by HRA Pharma. SIGNIFOR (pasireotide), developed by Recordati, was the first drug specifically approved for Cushing's disease by the FDA in 2012, and also gained EU approval. Recordati also markets SIGNIFOR LAR, a long-acting injectable version approved by the FDA and Japan's PMDA in 2018. This formulation, administered intramuscularly once every four weeks, shares the same mechanism as SIGNIFOR but offers a different delivery route. Recordati is recognized as a leading player in this therapeutic area. Another key product from the company is ISTURISA (osilodrostat), the first FDA-approved oral drug that inhibits cortisol production by targeting the enzyme 11β-hydroxylase (CYP11B1), which is involved in the final step of cortisol synthesis. Its oral administration makes it more convenient for patients compared to injectable therapies. ISTURISA is also being studied for pediatric use in an ongoing Phase II trial (NCT03708900). RECORLEV, originally developed by Strongbridge Biopharma and later acquired by Xeris Pharmaceuticals, was approved by the FDA in 2021. It is indicated for adults with endogenous Cushing's syndrome who are not surgical candidates or did not achieve a cure through surgery. Unlike ISTURISA, RECORLEV works by inhibiting cortisol production at multiple points in the steroidogenesis pathway, offering a distinct mechanism of action. To know more about Cushing's disease treatment guidelines, visit @ Cushing's Disease Management Cushing's Disease Pipeline Therapies and Key Companies Relacorilant (CORT125134): Corcept Therapeutics Clofutriben (SPI-62): Sparrow Pharmaceuticals Atumelnant (CRN04894): Crinetics Pharmaceuticals Lu AG13909: H. Lundbeck ST-002: Stero Therapeutics Discover more about Cushing's disease drugs in development @ Cushing's Disease Clinical Trials Cushing's Disease Market Dynamics The Cushing's disease market dynamics are expected to change in the coming years. Although Cushing's disease is generally considered a rare condition, growing awareness and recent advances in research suggest that diagnoses may rise in the future; despite a seemingly crowded market, many current hypercortisolism treatments fail to normalize cortisol levels effectively and pose significant side effects highlighting a substantial opportunity for innovation, especially given the limited therapies specifically targeting Cushing's disease and the potential of newer oral alternatives like ISTURISA and RECORLEV to offer greater flexibility compared to SC and intramuscular options such as SIGNIFOR and SIGNIFOR LAR. Furthermore, potential therapies are being investigated for the treatment of Cushing's disease, and it is safe to predict that the treatment space will significantly impact the Cushing's disease market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Cushing's disease market in the 7MM. However, several factors may impede the growth of the Cushing's disease market. Due to non-specific guidelines and recommendations, patients with Cushing's disease often face suboptimal care, delayed diagnosis, and inconsistent treatment approaches, further compounded by the diagnostic complexity, frequent misdiagnosis, and symptom overlap with more common conditions, which leave many undiagnosed or untreated; while existing therapies such as ketoconazole and metyrapone approved in Europe based on limited retrospective data and used off-label in the US despite high costs are widely trusted and entrenched in clinical practice, posing a significant barrier to the adoption of emerging therapies. Moreover, Cushing's disease treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Cushing's disease market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Cushing's disease market growth. Scope of the Cushing's Disease Market Report Therapeutic Assessment: Cushing's Disease current marketed and emerging therapies Cushing's Disease Market Dynamics: Key Market Forecast Assumptions of Emerging Cushing's Disease Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Cushing's Disease Market Access and Reimbursement Download the report to understand which factors are driving Cushing's disease market trends @ Cushing's Disease Market Trends Table of Contents Related Reports Cushing's Syndrome Market Cushing's Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Cushing's syndrome companies including Corcept Therapeutics, Crinetics Pharmaceuticals, Cyclacel Pharmaceuticals, Sparrow Pharmaceuticals, Stero Therapeutics, among others. Cushing's Syndrome Pipeline Cushing's Syndrome Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Cushing's syndrome companies, including Corcept Therapeutics, Crinetics Pharmaceuticals, Cyclacel Pharmaceuticals, Sparrow Pharmaceuticals, Stero Therapeutics, among others. Endogenous Cushing's Syndrome Market Endogenous Cushing's Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key endogenous Cushing's syndrome companies including Corcept Therapeutics, Crinetics Pharmaceuticals, Sparrow Pharmaceuticals, among others. Endogenous Cushing's Syndrome Pipeline Endogenous Cushing's Syndrome Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key endogenous Cushing's syndrome companies, including Corcept Therapeutics, Crinetics Pharmaceuticals, Sparrow Pharmaceuticals, among 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 . 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- 28-week data from the Phase 2b VANTAGE PBC study highlights statistically significant improvements in pruritus and numeric improvements in fatigue, the two most burdensome symptoms of PBC - Seventy percent of patients treated with volixibat experienced a ≥50% reduction in serum bile acids - Volixibat treated patients experienced reductions in IL-31 FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today presented new data from its Phase 2b VANTAGE study at the European Association for the Study of the Liver (EASL) meeting in Amsterdam, the Netherlands. The VANTAGE study is evaluating volixibat in patients with cholestatic pruritus caused by primary biliary cholangitis (PBC). “The extended VANTAGE analysis provides encouraging insights into the potential impact that volixibat may have in treating the most severe and bothersome symptoms for patients living with PBC, including pruritus and fatigue,” said Joanne Quan, MD, chief medical officer at Mirum. “We are also encouraged by the reductions seen in IL-31 in conjunction with sBA reductions, showing the potential for volixibat treatment to help alter the inflammatory condition present in PBC.” Data from the interim analysis of VANTAGE were highlighted with new analyses through week 28. Patients were randomized to receive volixibat 20mg BID, 80mg BID, or placebo BID. The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale, from baseline to Week 17-Week 28. Volixibat treatment led to rapid (as early as week 1) and sustained reductions in ItchRO with the volixibat combined dose group achieving a statistically significant 3.78-point reduction from Baseline (p<0.0001) and a placebo-adjusted response of 2.51 (p=0.0004). Each volixibat dose achieved statistically significant and similar responses (20mg BID: -2.4 [P=0.0039]; 80mg BID: -2.6 [P=0.0010]). The analysis also provided data across secondary endpoints showing reductions in serum bile acids (sBA) and quality of life (QoL) measures. Overall, 70% of patients who received volixibat achieved ≥50% reduction in sBA. Further, the data showed improvement from baseline in health-related quality of life, including fatigue and sleep, for patients treated with volixibat. Reductions from baseline were observed in inflammatory biomarker IL-31 in patients treated with volixibat. No new safety signals were observed, and adverse events were similar between the 20 mg and 80 mg treatment groups. The most common adverse event was diarrhea (77%) with all cases mild to moderate; one case resulted in discontinuation. Given the similar results with both efficacy and safety between the 20mg BID and 80mg doses BID, the 20mg BID dose was selected for the confirmatory portion of the VANTAGE study. To view the presentation, please visit the Publications & Presentations on Mirum’s website. About the VANTAGE Study VANTAGE is a randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of volixibat in patients with pruritus caused by primary biliary cholangitis (PBC). Patients were randomized to receive volixibat 20mg BID (n=10), 80mg BID (n=10), or placebo BID (n=11). The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale (an 11 point scale where 0 = no itch and 10 = worst itch imaginable), from baseline to Week 17-Week 28. VANTAGE is designed as a two-part study: the first part is intended to confirm dose (completed) and the second intended to be the confirmatory portion (currently enrolling patients). In the first part of the study, patients were randomized to receive either volixibat 20mg BID, 80mg BID, or placebo BID; the second part of the study randomized patients to receive either volixibat 20mg BID (the selected dose based on part one) or placebo BID. About Primary Biliary Cholangitis Primary Biliary Cholangitis (PBC) is a chronic, progressive, immune-mediated condition leading to the destruction of small intrahepatic bile ducts. The disease impacts an estimated 230,000 patients in the United States and the European Union, with women predominantly affected (a sex ratio of 9:1). Key clinical features and symptoms include cholestatic pruritus, fatigue, sicca syndrome, abdominal pain, cirrhosis, and hepatocellular carcinoma. Cholestatic pruritus (itch) and fatigue are two of the most debilitating symptoms and greatly affect overall health-related quality of life (HRQoL). Cholestatic pruritus can affect up to 80% of individuals and is thought to result in part from accumulation of toxic bile acids. About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases, in both liquid and tablet formulations. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. U.S. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. LIVMARLI is available in oral solution or tablet formulations and is taken by mouth 30 minutes before a meal. For Alagille syndrome, LIVMARLI is taken one time each day in the morning. For PFIC, LIVMARLI is taken two times each day. If you take the oral solution, use the oral dosing dispenser to measure your dose. US Prescribing Information EU SmPC Canadian Product Monograph About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, data from Mirum’s volixibat studies, including the VANTAGE study of volixibat in patients with pruritus due to PBC and such implications for patients in real world settings. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “will,” “could,” “can,” “would,” “potential,” “hope,” “opportunity,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on February 26, 2025, and subsequent filings with the U.S. Securities and Exchange Commission, which are available at www.sec.gov. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Media Contact: Erin Murphy media@mirumpharma.com Investor Contact: Andrew McKibben ir@mirumpharma.com