Mirum Pharmaceuticals (MIRM) has upcoming FDA regulatory milestones for Maralixibat. The table below outlines estimated target dates and event types for these pending regulatory actions.
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
volixibat, brelovitug, Maralixibat, and MRM-3379. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
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 4, 2026
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc announced the primary endpoint was met in the VISTAS Phase 2b study evaluating volixibat, an investigational oral ileal bile acid transporter (IBAT) inhibitor, in patients with primary sclerosing cholangitis (PSC).
AI Summary
Mirum Pharmaceuticals reported that the VISTAS Phase 2b study of volixibat, an investigational oral ileal bile acid transporter (IBAT) inhibitor for primary sclerosing cholangitis (PSC), met its primary endpoint. Patients experienced a statistically significant and clinically meaningful reduction in cholestatic pruritus: a 2.72‑point drop from baseline and a 1.64‑point placebo‑adjusted improvement (p<0.0001).
The company said full study results will be presented in a late‑breaking oral session at the EASL International Liver Congress on May 30, 2026 (2:15 p.m. CEST). Mirum also is holding a conference call today, May 4, at 8:30 a.m. ET to discuss topline findings from VISTAS and other recent clinical readouts.
This outcome supports continued development of volixibat in cholestatic liver disease and will inform the company’s next clinical and regulatory steps for PSC.Read Announcement
- Drug:
- volixibat
- Announced Date:
- September 8, 2025
- Indication:
- For treatment of pruritus in PBC
Announcement
Mirum Pharmaceuticals, Inc announced the completion of enrollment in the Phase 2b VISTAS study of volixibat, an investigational oral IBAT inhibitor, for the treatment of cholestatic pruritus in patients with primary sclerosing cholangitis (PSC).
AI Summary
Mirum Pharmaceuticals announced it has finished enrolling patients in the Phase 2b VISTAS study of volixibat, an oral IBAT inhibitor for cholestatic itch in primary sclerosing cholangitis (PSC). The trial met its goals at a blinded interim analysis in 2024 and aims to report topline data in the second quarter of 2026.
Dr. Joanne Quan, Mirum’s chief medical officer, said this milestone reflects the efforts of patients, investigators, and the company to fight PSC symptoms like itch and fatigue. Ricky Safer of PSC Partners Seeking a Cure added that completing enrollment brings new hope to those living with PSC, a disease with no approved therapies.
The VISTAS study is a global, randomized, double-blind, placebo-controlled trial over 28 weeks. Its primary endpoint is itch reduction measured by the Adult ItchRO scale. Secondary assessments include fatigue, serum bile acids, and safety. Successful results could pave the way for the first dedicated treatment for cholestatic pruritus in PSC.
Read Announcement- 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 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:
- 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:
- 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
Brelovitug FDA Regulatory Events
Brelovitug is a drug developed by Mirum Pharmaceuticals for the following indication: treatment of chronic hepatitis delta virus (HDV).
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:
- brelovitug
- Announced Date:
- April 27, 2026
- Indication:
- treatment of chronic hepatitis delta virus (HDV)
Announcement
Mirum Pharmaceuticals, Inc announced the primary endpoint was met in the Phase 2b portion of the AZURE-1 study evaluating brelovitug, an investigational monoclonal antibody designed to bind hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV).
AI Summary
Mirum Pharmaceuticals announced the primary endpoint was met in the Phase 2b portion of the AZURE-1 study of brelovitug, an investigational monoclonal antibody that binds hepatitis B surface antigen (HBsAg) for treatment of chronic hepatitis delta virus (HDV). The analysis included the first 53 patients evaluated at Week 24.
At Week 24, brelovitug showed strong antiviral activity and biochemical improvement: 100% of patients in the 300 mg once-weekly arm and 75% in the 900 mg once-every-four-weeks arm achieved the combined virologic response and ALT normalization, versus 0% in the delayed treatment arm. The treatment was reported to be well tolerated, with favorable safety and tolerability across dose groups.
Full Phase 2b results will be shown in a late-breaking poster at the EASL Congress (May 27–30, 2026). Topline Phase 3 data from AZURE-1 and AZURE-4 are expected in the second half of 2026.
Read Announcement- Drug:
- brelovitug
- Announced Date:
- March 5, 2026
- Indication:
- treatment of chronic hepatitis delta virus (HDV)
Announcement
Mirum Pharmaceuticals, Inc announced completion of enrollment in the Phase 3 AZURE-1 study and completion of screening in the Phase 3 AZURE-4 study, both evaluating brelovitug for the treatment of chronic hepatitis delta virus (HDV).
AI Summary
Mirum Pharmaceuticals announced it has completed enrollment in the Phase 3 AZURE-1 study and finished screening in the Phase 3 AZURE-4 study, both testing brelovitug for chronic hepatitis delta virus (HDV).
Topline results from AZURE-1 and AZURE-4 are expected in the second half of 2026. Together these two studies will form the basis of Mirum’s U.S. Biologics License Application (BLA), with a potential BLA submission and U.S. launch planned in 2027.
The global AZURE program is a registrational Phase 3 effort assessing combined virologic response and ALT normalization at Week 24 as the primary endpoint, per accelerated approval guidance. Both trials include open-label extensions up to 96 weeks.
Brelovitug targets HDV, the severe form of viral hepatitis that occurs in people with hepatitis B and can cause fibrosis, cirrhosis, liver cancer and death. No HDV therapies are approved in the U.S.
Read Announcement
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:
- March 16, 2026
- Target Action Date:
- Q4 2026
- Estimated Target Date Range:
- October 1, 2026 - December 31, 2026
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc. announced that the Topline data expected in Q4 2026
AI Summary
Mirum Pharmaceuticals said topline data from the EXPAND Phase 3 study are expected in Q4 2026. The company has completed enrollment, and patient groups say the study brings hope for a new treatment option for children and adults living with biliary atresia and other rare cholestatic conditions that cause severe, persistent itch.
EXPAND is designed to support a potential label expansion of LIVMARLI into additional settings of cholestatic pruritus. The trial’s primary endpoint is change in pruritus severity from baseline to Week 20. Secondary endpoints include changes in serum bile acids and other markers of cholestatic liver disease. Topline results will indicate whether the study met its key goals and could influence clinical and regulatory decisions.
LIVMARLI (maralixibat) is Mirum’s therapy for some cholestatic conditions; it is not for patients with PFIC type 2 who have a severe BSEP defect and can cause side effects. The Q4 2026 topline readout will be closely watched by patients, clinicians, and investors.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- March 16, 2026
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced completion of enrollment in EXPAND, a Phase 3 randomized, double-blind, placebo-controlled study evaluating LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients aged 6 months or older with rare cholestatic liver diseases, including biliary atresia.
AI Summary
Mirum Pharmaceuticals announced it has completed enrollment in EXPAND, a Phase 3 randomized, double-blind, placebo-controlled study evaluating LIVMARLI® (maralixibat) for cholestatic pruritus. The trial enrolls patients aged 6 months and older with rare cholestatic liver diseases, including biliary atresia, and is designed to support a potential label expansion into additional settings of cholestatic itch.
The study’s primary endpoint is change in pruritus severity from baseline to Week 20. Secondary endpoints include changes in serum bile acids and other markers of cholestatic liver disease. Topline data from EXPAND are expected in the fourth quarter of 2026.
“Families affected by biliary atresia and other rare cholestatic conditions understand the relentless burden of itch,” said Jen Lau, Co-Founder and Executive Director of BARE, Inc. She added that completing enrollment brings hope that more patients may soon have access to a much-needed treatment option.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- February 5, 2026
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced that Health Canada has authorized the tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).
AI Summary
Mirum Pharmaceuticals announced that Health Canada has authorized the tablet formulation of LIVMARLI (maralixibat) for the treatment of cholestatic pruritus in people with Alagille syndrome (ALGS). The tablet complements the existing 9.5 mg/ml oral solution by offering another dosing option for patients who weigh 22 kg or more and can swallow tablets.
The company said the tablet gives older patients a convenient one‑tablet dose while the liquid formulation remains available for younger children, increasing dosing flexibility and supporting continuity of care as patients age. ALGS is a rare genetic condition that affects multiple organs, including the liver; bile duct problems can cause cholestasis and severe itching that disrupts sleep, growth and daily life.
Important safety notes: LIVMARLI is not for use in PFIC type 2 patients with a severe BSEP defect, and the drug can cause side effects. Patients and caregivers should discuss benefits and risks with their healthcare provider.
Read Announcement- Drug:
- Maralixibat
- Announced Date:
- November 7, 2025
- Indication:
- Alagille Syndrome
Announcement
Mirum Pharmaceuticals, Inc announced that multiple presentations featuring data from its LIVMARLI (maralixibat) and volixibat clinical programs will be shared during the American Association for the Study of Liver Diseases' (AASLD) The Liver Meeting®, taking place November 7-11, 2025, in Washington, D.C.
AI Summary
Mirum Pharmaceuticals announced that it will share new data from its LIVMARLI (maralixibat) and volixibat clinical programs at the American Association for the Study of Liver Diseases’ The Liver Meeting®, November 7–11, 2025, in Washington, D.C. The company’s presentations will highlight progress in treating rare adult and pediatric liver diseases.
An oral session will feature additional analyses from the VANTAGE trial showing volixibat’s impact on fatigue and sleep in adults with primary biliary cholangitis. Multiple poster abstracts will report real-world experience with maralixibat in primary sclerosing cholangitis, the clinical utility of comprehensive genetic testing in adult cholestasis, preliminary results on volixibat’s effects on IL-31 and bile acids in cholestatic patients, a physician survey on pruritus in PSC across four countries, and the impact of maralixibat on caregiver burden in Alagille syndrome and PFIC.
Mirum will also host a fireside discussion on diagnosing and managing progressive familial intrahepatic cholestasis, including a review of LIVMARLI clinical data. These presentations underscore Mirum’s dedication to advancing therapies that improve patient outcomes in rare liver diseases.
Read Announcement- 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
- Target Action Date:
- Q3 2024
- Estimated Target Date Range:
- July 1, 2024 - September 30, 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 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 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
MRM-3379 FDA Regulatory Events
MRM-3379 is a drug developed by Mirum Pharmaceuticals for the following indication: Fragile X 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:
- MRM-3379
- Announced Date:
- January 9, 2026
- Indication:
- Fragile X syndrome
Announcement
Enthorin Therapeutics, LLC announced that its licensing partner forMRM-3379(formerly ENT-3379), Mirum Pharmaceuticals, Inc n December, reported the initiation of the BLOOM Phase 2 clinical study evaluating MRM-3379 in Fragile X syndrome (FXS).
AI Summary
Enthorin Therapeutics announced that its licensing partner, Mirum Pharmaceuticals, reported in December the start of the BLOOM Phase 2 study testing MRM-3379 (formerly ENT-3379) in Fragile X syndrome (FXS). The study will evaluate the oral, brain-penetrant compound in people with FXS under Mirum’s development program.
MRM-3379 is a selective PDE4D inhibitor designed to boost cAMP signaling and restore neural signaling. The molecule was discovered by scientists at Dart Neuroscience, including current Enthorin team members, and completed Phase 1 at DNS. Preclinical work, including studies in FMR1 knockout mice, suggests MRM-3379 can improve cognition and reduce behavioral deficits across multiple domains.
Mirum’s move into patient trials represents the culmination of more than a decade of research by Enthorin and DNS. Enthorin acknowledged the clinicians and scientists who contributed to the discovery and development of MRM-3379. The program aims to address core biology underlying FXS.
Read Announcement
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