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181 News |  |
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- |||| Livmarli (maralixibat) / Mirum Pharma
Enrollment open: Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND) (clinicaltrials.gov) - Oct 16, 2024
P3, N=90, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Not yet recruiting --> Recruiting
- Livmarli (maralixibat) / Mirum Pharma
IMPROVEMENT IN SEQUELAE OF CHRONIC LIVER DISEASE IN PATIENTS WITH ALAGILLE SYNDROME TREATED WITH MARALIXIBAT () - Oct 15, 2024 - Abstract #AASLD2024AASLD_3198;
In addition, the first patient demonstrated resolution of esophageal varices and the second patient experienced a decreased appearance of xanthomas; both occurred in the absence of additional therapeutic interventions. It is promising that Maralixibat may affect progression and manifestations of chronic liver disease, including portal hypertension and xanthomas, that are often indications for liver transplantation.
- Livmarli (maralixibat) / Mirum Pharma
BILE ACID SUBSPECIES ARE CORRELATED WITH PRURITUS AND BILIRUBIN IMPROVEMENT IN PFIC PATIENTS TREATED WITH MARALIXIBAT: DATA FROM MARCH AND MARCH-ON () - Oct 15, 2024 - Abstract #AASLD2024AASLD_3189;
This pattern indicates either new BA synthesis in response to reduced BA levels in the liver or increased bacterial action on BAs in the colon. These data further elucidate the pathophysiology of how MRX may influence improvements in both pruritus and underlying liver health in patients with PFIC.
- Livmarli (maralixibat) / Mirum Pharma
THE RELATIONSHIP BETWEEN SERUM BILE ACIDS AND EVENT-FREE SURVIVAL FOLLOWING THE USE OF MARALIXIBAT FOR PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM MARCH/MARCH-ON () - Oct 15, 2024 - Abstract #AASLD2024AASLD_3185;
Consistent with sBA response thresholds from the NAPPED Consortium that are associated with EFS, individuals in MARCH/MARCH-ON who reduced sBA levels below the threshold did not have a clinically meaningful event whereas some individuals who had lower reductions in sBA experienced events. These data support the importance of sBA reduction in PFIC and the potential of maralixibat to facilitate this biochemical change.
- Livmarli (maralixibat) / Mirum Pharma
REAL-WORLD USE OF MARALIXIBAT IN BILIARY ATRESIA: A CASE SERIES () - Oct 15, 2024 - Abstract #AASLD2024AASLD_3173;
Maralixibat may be effective in patients with BA for the management of cholestatic pruritus, including in patients with significant pruritus and progressive liver disease. This data supports the need for larger studies to systematically evaluate the potential benefit of MRX for the treatment of cholestatic pruritus in BA.
- Livmarli (maralixibat) / Mirum Pharma
IMPACT OF MARALIXIBAT ON CAREGIVER BURDEN FOR PATIENTS WITH ALAGILLE SYNDROME AND PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS () - Oct 15, 2024 - Abstract #AASLD2024AASLD_3156;
This study provides early data of caregiver burden in caregivers of patients with ALGS including negative impact on sleep, productivity, employment and mental health. Further longitudinal data is needed to better quantify this impact and determine if MRX can mitigate this concern.
- Livmarli (maralixibat) / Mirum Pharma
IMPROVEMENTS IN PRURITUS ARE ASSOCIATED WITH IMPROVEMENTS IN GROWTH IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM THE MARCH-ON TRIAL () - Oct 15, 2024 - Abstract #AASLD2024AASLD_1430;
MARCH was a 26-week, randomized, placebo-controlled (PBO), Phase 3 trial of maralixibat (MRX), an ileal bile acid transporter (IBAT) inhibitor, that achieved its primary and key secondary endpoints of pruritus and sBA reduction for individuals across the broadest range of PFIC types studied... These consistent trends in growth for participants who received MRX and were pruritus responders indicate a potential disease-modifying effect of MRX treatment in PFIC.
- ||| Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: An Extension Study of Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Oct 2, 2024
P3, N=90, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
These consistent trends in growth for participants who received MRX and were pruritus responders indicate a potential disease-modifying effect of MRX treatment in PFIC. Trial completion date: Sep 2024 --> Dec 2024 | Trial primary completion date: Sep 2024 --> Dec 2024
- | Livmarli (maralixibat) / Mirum Pharma
Journal: Stereoselective Synthesis of Maralixibat via VO(acac)2/Schiff Base-Catalyzed Asymmetric Oxidation of Its Sulfide Intermediate. (Pubmed Central) - Sep 20, 2024
The stereoselective synthesis of maralixibat was achieved by harnessing the chiral transferring effect of the stereogenic R-sulfoxide functionality, which was obtained via the VO(acac)2/Schiff base-catalyzed asymmetric oxidation of a phenylthiophenol prochiral intermediate. The R-sulfoxide intermediate underwent a ring closure reaction to form the seven-membered ring core structure with the desired stereochemistry, ultimately ensuring the drug's exceptional isomeric purity and synthetic efficiency.
- |||||||| Livmarli (maralixibat) / Mirum Pharma
New P3 trial: Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND) (clinicaltrials.gov) - Aug 14, 2024
P3, N=90, Not yet recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
- || Review, Journal: Transporter Proteins as Therapeutic Drug Targets-With a Focus on SGLT2 Inhibitors. (Pubmed Central) - Jul 13, 2024
Probenecid can be considered to increase uric acid excretion in the urine mainly via the inhibition of urate transporter 1 (URAT1), and due to pharmacokinetic interactions involving organic anion transporters 1 and 3 (OAT1 and OAT3), it modifies renal excretion of penicillins or ciprofloxacin as well as nephrotoxicity of cidofovir. This review discusses clinically approved drugs that affect membrane/drug transporter function.
- ||| Livmarli (maralixibat) / Mirum Pharma
P3 data, Journal: Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. (Pubmed Central) - Jun 13, 2024
P3
Maralixibat improved pruritus and predictors of native liver survival in PFIC (eg, serum bile acids). Maralixibat represents a non-surgical, pharmacological option to interrupt the enterohepatic circulation and improve the standard of care in patients with PFIC.
- Livmarli (maralixibat) / Mirum Pharma
Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH): A randomized placebo-controlled phase 3 study () - Apr 9, 2024 - Abstract #AMCP2024AMCP_400;
- Livmarli (maralixibat) / Mirum Pharma
Improvements in serum bile acid levels are associated with improvements in key markers of liver health after maralixibat treatment in children with progressive familial intrahepatic cholestasis: data from the MARCH/MARCH-ON trials (Poster Area) - Apr 2, 2024 - Abstract #EASLILC2024EASL_ILC_967;
Reductions in sBA following use of MRX, as anticipated by the drug's mechanism of action, are correlated with reductions in important biomarkers of liver disease like bilirubin, which has been shown to be associated with long-term clinical outcomes. These results demonstrate the potential for MRX to have disease-modifying effects in the treatment of PFIC.
- Livmarli (maralixibat) / Mirum Pharma
Maralixibat improves growth in patients with progressive familial intrahepatic cholestasis: data from the MARCH/MARCH-ON trials (Poster Area) - Apr 2, 2024 - Abstract #EASLILC2024EASL_ILC_965;
These results demonstrate the potential for MRX to have disease-modifying effects in the treatment of PFIC. These consistent trends in growth for MRX-treated PFIC patients of various types represents the first report of sustained improvements in weight, as well as improvements in height, with the use of an IBAT inhibitor indicating a potential disease-modifying effect of MRX treatment in PFIC.
- Livmarli (maralixibat) / Mirum Pharma
Clinical benefits with maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: data from the MERGE study (Poster Area) - Apr 2, 2024 - Abstract #EASLILC2024EASL_ILC_964;
P2
In this unmatched cohort, the benefit of MRX in ALGS patients, including both improvements in clinical outcomes and sBA, persist through 7 years of treatment. No new safety concerns were identified in the long-term.
- | Livmarli (maralixibat) / Mirum Pharma
Trial completion date: RISE: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (clinicaltrials.gov) - Mar 15, 2024
P2, N=12, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
No new safety concerns were identified in the long-term. Trial completion date: May 2024 --> Dec 2024
- ||| Livmarli (maralixibat) / Mirum Pharma
Trial completion, Trial completion date: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Mar 12, 2024
P2, N=75, Completed, Sponsor: Mirum Pharmaceuticals, Inc.
Trial completion date: May 2024 --> Dec 2024 Active, not recruiting --> Completed | Trial completion date: Mar 2025 --> Feb 2024
- | Livmarli (maralixibat) / Mirum Pharma
Journal, Real-world evidence, Real-world: Real-world experience of maralixibat in Alagille syndrome: Novel findings outside of clinical trials. (Pubmed Central) - Feb 9, 2024
Active, not recruiting --> Completed | Trial completion date: Mar 2025 --> Feb 2024 Maralixibat may be effective and well tolerated in patients with ALGS in broader clinical contexts than previously reported.
- |||||| Livmarli (maralixibat) / Mirum Pharma
New trial: Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP) (clinicaltrials.gov) - Jan 5, 2024
P=N/A, N=50, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
- | Livmarli (maralixibat) / Mirum Pharma, Takeda
Journal, Real-world evidence, Real-world: Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA. (Pubmed Central) - Dec 26, 2023
This study demonstrates a novel application of a robust statistical method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible, providing wide application for rare diseases. This comparison with real-world natural history data suggests that maralixibat improves EFS in patients with ALGS.
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Long-Term Maintenance of Response and Improved Liver Heath With Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC): 2-Year Data From the MARCH-ON Study (Sancy - Hilton Antwerp Old Town) - Dec 19, 2023 - Abstract #BWG2024BWG_112;
Conclusions Significant and sustained improvements in pruritus, sBA, TB, and growth are observed with up to 2 years of MRX treatment across the broadest range of genetic PFIC types studied to date. These data suggest overall improved liver health with MRX treatment which can be maintained long-term.
- || Livmarli (maralixibat) / Mirum Pharma, Takeda
Trial completion date, Trial primary completion date: MERGE: MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (clinicaltrials.gov) - Dec 7, 2023
P2, N=52, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
These data suggest overall improved liver health with MRX treatment which can be maintained long-term. Trial completion date: Jan 2024 --> Apr 2024 | Trial primary completion date: Jan 2024 --> Apr 2024
- | Livmarli (maralixibat) / Mirum Pharma, Takeda
Journal: Predictors of 6-year event-free survival in alagille syndrome patients treated with maralixibat, an ileal bile acid transporter inhibitor. (Pubmed Central) - Nov 27, 2023
Improvement in pruritus by 48 weeks, and lower W48 bilirubin and serum bile acid levels were associated with fewer events. These data may help identify potential markers of disease progression for ALGS patients treated with MRX.
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Trial primary completion date: A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (clinicaltrials.gov) - Nov 7, 2023
P3, N=7, Active, not recruiting, Sponsor: Takeda
These data may help identify potential markers of disease progression for ALGS patients treated with MRX. Trial primary completion date: Jul 2025 --> Oct 2023
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment closed, Trial completion date: A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Nov 7, 2023
P3, N=5, Active, not recruiting, Sponsor: Takeda
Trial primary completion date: Jul 2025 --> Oct 2023 Completed --> Active, not recruiting | Trial completion date: Sep 2023 --> May 2025
- || Livmarli (maralixibat) / Mirum Pharma
Trial primary completion date: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Oct 6, 2023
P2, N=72, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Completed --> Active, not recruiting | Trial completion date: Sep 2023 --> May 2025 Trial primary completion date: Aug 2023 --> Nov 2023
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Trial completion, Trial completion date, Trial primary completion date: A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Oct 2, 2023
P3, N=5, Completed, Sponsor: Takeda
Trial primary completion date: Aug 2023 --> Nov 2023 Active, not recruiting --> Completed | Trial completion date: Jul 2025 --> Sep 2023 | Trial primary completion date: Jul 2025 --> Sep 2023
- || Livmarli (maralixibat) / Mirum Pharma, Takeda
Review, Journal: Management of adults with Alagille syndrome. (Pubmed Central) - Aug 16, 2023
This review is aimed at providing a comprehensive review of ALGS to adult clinicians who will take over the medical care of these patients following transition, with particular focus on certain aspects of the condition that require lifelong surveillance. We also provide a diagnostic framework for adult patients with suspected ALGS and highlight key aspects to consider when determining eligibility for LT in patients with this syndrome.
- Livmarli (maralixibat) / Mirum Pharma, Takeda, Bylvay (odevixibat) / Ipsen
Ileal Bile Acid Transporter Inhibition for Treatment of Pruritis in Progressive Familial Intrahepatic Cholestasis (Exhibit Hall) - Jul 29, 2023 - Abstract #ACG2023ACG_3066;
Additionally, novel therapies like IBAT inhibitors, specifically Odevixibat and Maralixibat, which received FDA approval in 2021, should be considered. Physicians caring for patients with this condition should be aware of these novel drug classes' benefits and side effects.
- |||| Livmarli (maralixibat) / Mirum Pharma
Clinical, Video: In a Healio video exclusive, Pam Vig, PhD, of @mirumpharma, highlights new data on #Livmarli for the treatment of patients with #pruritis due to #PFIC and #Alagillesyndrome, which were presented at #EASLCongress. https://t.co/8MFuFOLXXv (Twitter) - Jun 26, 2023
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment closed: A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (clinicaltrials.gov) - Jun 8, 2023
P3, N=7, Active, not recruiting, Sponsor: Takeda
Physicians caring for patients with this condition should be aware of these novel drug classes' benefits and side effects. Recruiting --> Active, not recruiting
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment closed: A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Jun 8, 2023
P3, N=5, Active, not recruiting, Sponsor: Takeda
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- Livmarli (maralixibat) / Mirum Pharma
Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: data from MARCH-PFIC (Immune mediated & cholestatic diseases) - May 31, 2023 - Abstract #EASLILC2023EASL_ILC_2533;
- || Livmarli (maralixibat) / Mirum Pharma
Enrollment closed: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - May 31, 2023
P2, N=72, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- | Livmarli (maralixibat) / Mirum Pharma
Journal: Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study. (Pubmed Central) - May 19, 2023
P2
These results support further investigation of IBAT inhibitors for adults with PSC-associated pruritus. ClinicalTrials.gov: NCT02061540.
- | Livmarli (maralixibat) / Mirum Pharma
Enrollment closed, Trial completion date, Trial primary completion date: RISE: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (clinicaltrials.gov) - May 19, 2023
P2, N=12, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov: NCT02061540. Recruiting --> Active, not recruiting | Trial completion date: Aug 2023 --> May 2024 | Trial primary completion date: Mar 2023 --> Jul 2023
- Livmarli (maralixibat) / Mirum Pharma
Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): data from the MARCH-ON study (Poster session) - May 17, 2023 - Abstract #EASLILC2023EASL_ILC_2419;
- Livmarli (maralixibat) / Mirum Pharma
Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: data from MARCH-PFIC (Stolz 1) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_2343;
MRX is the only IBAT inhibitor to demonstrate significant decreases in TB/DB compared to PBO in children with PFIC and across PFIC types. 40% of MRX patients with abnormal Baseline bilirubin achieved normalization vs none in the PBO group, suggesting that MRX may yield clinically meaningful improvements in liver health in patients with PFIC.
- Livmarli (maralixibat) / Mirum Pharma
Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: data from MARCH-PFIC (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_531;
MRX was well-tolerated, with GI effects being the most frequent event but generally mild and self-limiting. FSV deficiency and bilirubin increase were more frequently seen in the PBO group.
- Livmarli (maralixibat) / Mirum Pharma
Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: data from MARCH-PFIC (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_530;
MRX was associated with complete or near-complete resolution of pruritus in the majority of patients with PFIC and the effect was independent of how it was measured, or who made the assessments. Changes with pruritus were strongly correlated with changes in sleep, suggesting that use of MRX may yield meaningful improvements in this domain of quality of life.
- Livmarli (maralixibat) / Mirum Pharma
Impact of maralixibat on cholestatic pruritus in adults aged 16 years and older with Alagille syndrome (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_529;
MRX was effective, durable and well tolerated in ALGS patients aged 16 years and older, providing critical data for patients who transition to adulthood while on therapy. These data support an ongoing role for treatment in adults with ALGS that survive with their native livers into adulthood.
- Livmarli (maralixibat) / Mirum Pharma
Maralixibat improves growth in patients with Alagille syndrome: A 4-year analysis () - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_138;
Increased catch-up height was also seen in patients that achieved lower sBA with MRX, suggesting an improvement in bile acid homeostasis may be a factor. Further analyses are needed comparing growth trajectories in MRX-treated patients to a natural history cohort of patients with ALGS to fully understand the attributability of MRX.
- Livmarli (maralixibat) / Mirum Pharma
Maralixibat-treated patients with Alagille syndrome demonstrate event-free survi30 year in a natural history comparison with patients with from the GALA database: Application of real-world evidence analytics () - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_70;
This RWE analysis provides a potential method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible. Limitations will always be present given lack of prospective conduct and inherent biases, though sensitivity analyses can help mitigate and aid interpretation.
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment open: A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (clinicaltrials.gov) - Jan 18, 2023
P3, N=5, Recruiting, Sponsor: Takeda
Limitations will always be present given lack of prospective conduct and inherent biases, though sensitivity analyses can help mitigate and aid interpretation. Not yet recruiting --> Recruiting
- Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment open: A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Jan 17, 2023
P3, N=9, Recruiting, Sponsor: Takeda
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting
- || Livmarli (maralixibat) / Mirum Pharma, Bylvay (odevixibat) / Albireo
Retrospective data, Review, Journal: Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis. (Pubmed Central) - Dec 24, 2022
The efficacy of IBAT inhibitors across current trials was noted. Future trials may focus on the optimization of dosing regimens, considering gastrointestinal side effects and drug-induced ALT elevation in AGLS patients.
- Livmarli (maralixibat) / Mirum Pharma
SAFETY AND TOLERABILITY CHARACTERIZATION OF MARALIXIBAT IN INFANTS WITH ALGS FROM 2 MONTHS OF AGE: INTERIM RESULTS FROM THE RISE STUDY () - Nov 2, 2022 - Abstract #AASLD2022AASLD_2409;
Future trials may focus on the optimization of dosing regimens, considering gastrointestinal side effects and drug-induced ALT elevation in AGLS patients. MRX was well-tolerated, with minimal to no absorption in infants with ALGS <1 year of age, indicating that MRX may be used in children as young as 2 months of age.
- Livmarli (maralixibat) / Mirum Pharma
EFFICACY AND SAFETY OF MARALIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (MARCH-PFIC): A RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY (Ballroom ABC) - Nov 2, 2022 - Abstract #AASLD2022AASLD_2389;
This trial is the largest interventional study conducted in this rare genetic disease. This study incorporates five PFIC types, some of which have not previously been studied in a clinical trial of an IBATi and will be presented for the first time.
- | Livmarli (maralixibat) / Mirum Pharma
Biomarker, Journal: Serum Bile Acid Profiling and Mixed Model Analysis Reveal Biomarkers Associated with Pruritus Reduction in Maralixibat-Treated Patients with BSEP Deficiency. (Pubmed Central) - Oct 28, 2022
Notably, the trajectory of serum total and individual BA species and 7α-C4 were significantly associated with ItchRO[Obs] score (p < 0.05). These results reveal that beyond simple total sBA concentrations, specific changes to the BA metabolome are associated with pruritus reduction in patients with BSEP deficiency, thus providing further insight into causal relationship of bile acids and pruritus.