This section highlights FDA-related milestones and regulatory updates for drugs developed by Mirum Pharmaceuticals (MIRM).
Over the past two years, Mirum Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Maralixibat and volixibat. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Maralixibat - FDA Regulatory Timeline and Events
Maralixibat is a drug developed by Mirum Pharmaceuticals for the following indication: Alagille Syndrome.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Maralixibat
- Announced Date:
- April 14, 2025
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
AI Summary
Mirum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI® (maralixibat) to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). This approval provides an important alternative to the existing liquid formulation, offering greater convenience for older patients who can now take a single tablet per dose. The new tablet formulation adds flexibility to treatment choices, as younger patients may still benefit from liquid dosing. Mirum’s president, Peter Radovich, emphasized that this approval not only expands treatment options but also aims to positively impact patients’ quality of life by making the medication easier to use. The new formulation is planned to be available in June, potentially offering a more streamlined therapy for those managing these rare liver conditions.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- July 8, 2024
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients three months of age and older.
AI Summary
Mirum Pharmaceuticals recently announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older. This approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), which recognized that LIVMARLI offers significant clinical benefits compared with current therapies. Clinical data from the Phase 3 MARCH study, which involved 93 patients with various PFIC types, revealed meaningful improvements such as marked reductions in pruritus and serum bile acid levels.
This decision is an important milestone for families affected by PFIC in Europe, as LIVMARLI not only addresses the symptoms but can also contribute to better overall liver health. The authorization underscores the commitment to advancing treatment options for this rare liver disease and brings new hope to young patients and their families.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- May 31, 2024
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc. announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older.
AI Summary
Mirum Pharmaceuticals, Inc. announced that the European Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion for LIVMARLI® (maralixibat) oral solution in treating Progressive Familial Intrahepatic Cholestasis (PFIC) in patients aged three months and older. This decision is based on data from the Phase 3 MARCH study, which showed a highly significant reduction in itch severity compared to placebo across all PFIC types. The CHMP concluded that LIVMARLI offers significant clinical benefits over the current approved therapy. Additionally, the Committee for Orphan Medicinal Products (COMP) has recommended maintaining its Orphan Drug Designation for LIVMARLI, highlighting its potential value for a rare disease. A decision from the European Commission on marketing authorization is expected in the third quarter of 2024.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- May 31, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc. announced Following CHMP and COMP opinions, a decision by the European Commission is expected in the third quarter of 2024.
AI Summary
Mirum Pharmaceuticals announced that, following positive opinions from both the CHMP and COMP, a decision from the European Commission is expected in the third quarter of 2024 regarding LIVMARLI® (maralixibat) for treating Progressive Familial Intrahepatic Cholestasis (PFIC). The CHMP reviewed data from the Phase 3 MARCH study, which showed a highly significant reduction in pruritus severity in PFIC patients compared to placebo and highlighted the treatment's improved benefits over existing therapies. Additionally, the COMP recommended maintaining the drug’s Orphan Drug Designation, underscoring its potential for addressing this severe, rare liver disease. If approved, LIVMARLI could offer a new therapeutic option for PFIC patients as young as three months old, representing a major step forward in patient care in Europe.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- May 29, 2024
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced that new analyses from long-term treatment with LIVMARLI will be presented at the European Association for the Study of the Liver (EASL) Annual Congress taking place June 5-8, 2024, in Milan, Italy.
AI Summary
Mirum Pharmaceuticals, Inc. announced that new analyses from long-term treatment with LIVMARLI will be showcased at the European Association for the Study of the Liver (EASL) Annual Congress in Milan, Italy from June 5-8, 2024. The presented data comes from studies on patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) and highlights sustained clinical benefits. One study shows that patients with ALGS continue to experience lasting improvements even after seven years of treatment, while another report details enhancements in key liver markers and growth in patients with PFIC. These presentations, scheduled during the poster sessions on June 6, aim to underline the long-term efficacy of LIVMARLI (maralixibat) and its potential to improve the quality of life for children suffering from these rare liver diseases.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- May 18, 2024
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced data presented during the 56th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting which took place this week in Milan, Italy. Data from LIVMARLI® (maralixibat) oral solution clinical studies and real-world settings in Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) were presented in oral and poster presentations during the meeting.
Read Announcement
volixibat - FDA Regulatory Timeline and Events
volixibat is a drug developed by Mirum Pharmaceuticals for the following indication: For treatment of pruritus in PBC.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- volixibat
- Announced Date:
- May 9, 2025
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc. 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.
AI Summary
Mirum Pharmaceuticals, Inc. presented new data from its Phase 2b VANTAGE study at the European Association for the Study of the Liver meeting in Amsterdam. The study focuses on the use of volixibat in patients with cholestatic pruritus due to primary biliary cholangitis (PBC). Findings from the 28-week analysis show that volixibat led to significant improvements in patients’ itch scores, reducing pruritus rapidly and in a sustained manner. In addition, there were numeric improvements in fatigue, one of the most challenging symptoms for PBC patients. The results highlighted that 70% of patients treated with volixibat achieved a reduction of at least 50% in serum bile acids. The treatment also lowered levels of the inflammatory biomarker IL-31. No new safety concerns were reported, and the safety profile was similar between the tested doses, reinforcing the potential of volixibat as an effective treatment option for PBC symptoms.
Read Announcement- Drug:
- volixibat
- Announced Date:
- April 28, 2025
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc announced that it will present data at three upcoming medical congresses. Digestive Disease Week (DDW) will be held in San Diego, May 3-6, 2025.
AI Summary
Mirum Pharmaceuticals, Inc. announced plans to present new research data at three upcoming medical congresses. The company will share findings from its LIVMARLI and volixibat studies through both oral and poster presentations. One key event is Digestive Disease Week (DDW) in San Diego, scheduled for May 3–6, 2025, where researchers will present interim data from the Phase 2b VANTAGE study. This analysis focuses on improvements in cholestatic pruritus, fatigue, and serum bile acid levels in patients with primary biliary cholangitis.
Other presentations are set for the European Association for the Study of the Liver (EASL) congress in Amsterdam and the 57th European Society for Paediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting in Helsinki. The data showcase Mirum’s commitment to advancing treatment options for cholestatic liver diseases, with positive early results emphasizing both symptom reduction and overall patient improvements.
Read Announcement- Drug:
- volixibat
- Announced Date:
- October 10, 2024
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc announced that The confirmatory portion of the study is ongoing with completion of enrollment expected in 2026.
AI Summary
Mirum Pharmaceuticals, Inc recently updated on its ongoing Phase 2b VANTAGE study, which is testing volixibat as a treatment for severe itching (pruritus) in patients with primary biliary cholangitis (PBC). The confirmatory portion of the study continues, with enrollment expected to be completed in 2026. This part of the trial is designed to further confirm the promising results observed in the interim analysis, reinforcing the potential of volixibat to significantly relieve the debilitating itch associated with PBC.
Mirum emphasizes that completing enrollment is a critical step toward gathering solid data on how well volixibat works. The company remains dedicated to advancing its research to offer new therapeutic options that could greatly improve the quality of life for patients suffering from this rare liver disease.
Read Announcement - Drug:
- volixibat
- Announced Date:
- October 10, 2024
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). .
AI Summary
Mirum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). This designation was based on a positive interim analysis from the Phase 2b VANTAGE study, which showed statistically significant improvements in itch severity compared to placebo.
The promising results underline the potential of volixibat to ease one of the most challenging symptoms of PBC. The confirmatory part of the study is still underway, with enrollment expected to be completed in 2026. The FDA’s designation is intended to expedite development and review processes for drugs that show serious benefits over current therapies, aiming to bring relief to patients suffering from PBC-related pruritus as quickly as possible.
Read Announcement- Drug:
- volixibat
- Announced Date:
- June 17, 2024
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc announced interim results from two Phase 2b studies evaluating volixibat, an oral ileal bile acid transporter (IBAT) inhibitor in patients with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC).
AI Summary
Mirum Pharmaceuticals announced promising interim results from two Phase 2b studies testing volixibat, an oral IBAT inhibitor, in patients with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC). In the VANTAGE study for PBC, patients experienced a significant reduction in pruritus, with an average 3.8-point improvement from baseline and a 2.3-point greater reduction than the placebo group (p=0.0026). Additionally, 75% of patients treated with volixibat achieved more than a 50% decrease in serum bile acids, and there was notable improvement in fatigue scores at week 16.
The VISTAS study for PSC also exceeded its efficacy threshold, allowing the continuation of the trial. No new safety concerns were noted, with diarrhea being the most common, mostly mild cases. These results suggest volixibat may offer a new treatment option for reducing the symptoms of these liver diseases.
Read Announcement
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