– Seven abstracts highlighting ALGS and PFIC knowledge, together with three oral displays
– Lengthy-term extension knowledge from the PFIC Part 3 examine are introduced on the plenary session and chosen from the best scoring abstracts
FOSTER CITY, Calif.–(BUSINESS WIRE)–Mirum Prescription drugs, Inc. (NASDAQ: MIRM) in the present day introduced plans for this 12 months’s 56th European Society of Pediatrics, Gastroenterology, Hepatology and Diet (ESPGHAN) Knowledge introduced on the annual assembly in Milan, Italy for the week. Scientific examine knowledge on LIVMARLI ® (maralixibat) oral resolution in addition to real-world setting knowledge on Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) had been introduced in oral and poster codecs throughout the assembly.
“We proceed to construct on robust proof that LIVMARLI has the potential to supply long-term advantages on key high quality of life and liver illness parameters for PFIC sufferers and enhance completely different genetic forms of PFIC,” stated lead researcher Pam Vig, Ph.D., scientific officer and director of analysis at Mirum. . Moreover, we’re happy to current knowledge exhibiting that kids handled with LIVMARLI are decreasing or discontinuing some anti-itch drugs and dietary dietary supplements.
Abstract 587: Maralixibat maintains long-term efficacy and improves liver well being in sufferers with progressive familial intrahepatic cholestasis: 2-year knowledge from the MARCH-ON examine
Plenary Session: High Rating Abstracts in Hepatology
Lectured by Professor Richard J. Thompson, MD, King’s Faculty London, UK
As much as two years after remedy with LIVMARLI, PFIC sufferers skilled important and sustained enhancements in pruritus severity, serum bile acid (sBA) ranges, whole bilirubin, and development. Comparable enhancements in pruritus and sBA had been noticed in sufferers initially randomized to placebo and receiving LIVMARLI within the open-label examine.
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Abstract 594: Maralixibat considerably reduces bilirubin in sufferers with progressive familial intrahepatic cholestasis: knowledge from the MARCH trial
Hosted by Lorenzo D’Antiga, MD, Pope Giovanni XXIII Hospital, Bergamo, Italy
Sufferers handled with LIVMARLI skilled important reductions in each whole and direct bilirubin in comparison with placebo. Forty p.c of sufferers who had irregular bilirubin at baseline achieved normalization within the LIVMARLI-treated sufferers in contrast with none within the placebo group. Moreover, the lower in bilirubin coincided with the lower in sBA.
Abstract 600: Impact of Mararixibat on Concomitant Drugs for the Remedy of Cholestatic Pruritus in Alagille Syndrome: Actual World Expertise
Offered by Jolan Terner-Rosenthal, Ph.D., Mirum Prescription drugs (NASDAQ: ), Inc. (Foster Metropolis, CA, USA)
Actual-world proof from 116 sufferers handled with LIVMARLI for at the least one 12 months confirmed that greater than one-third of sufferers had been capable of discontinue 1 concomitant antipruritic treatment and one-fifth of sufferers discontinued 2 drugs. Concomitant drug use was decreased throughout all drug varieties, suggesting that LIVMARLI can scale back polypharmacy burden throughout the first 12 months of remedy.
Different displays throughout ESPGHAN embody:
Abstract 592: Maralixibat considerably improves cholestatic pruritus in kids with genetically undiagnosed progressive familial intrahepatic cholestasis: knowledge from the MARCH trial
Lectured by Professor Richard J. Thompson, MD, King’s Faculty London, UK←
Abstract 599: Maralixibat improves cholestatic pruritus in kids with progressive familial intrahepatic cholestasis because of MDR3 deficiency: knowledge from the MARCH/MARCH-ON trial
Lectured by Professor Richard J. Thompson, MD, King’s Faculty London, UK
Abstract 595: Enchancment of pruritus with maralixibat is related to improved high quality of life in sufferers with progressive familial intrahepatic cholestasis: knowledge from the MARCH trial
Offered by Douglas B. Mogul, MD, PhD, Mirum Prescription drugs, Inc., Foster Metropolis, California, USA
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Abstract 597: Maralixibat improves cholestatic pruritus in kids with progressive familial intrahepatic cholestasis who’ve beforehand undergone biliary diversion surgical procedure: knowledge from the MARCH/MARCH-ON trial
Hosted by Lorenzo D’Antiga, MD, Pope Giovanni XXIII Hospital, Bergamo, Italy
To view the complete presentation, please go to the Publications part of the Mirum web site.
About LIVMARLI ® (maralixibat) Oral Resolution
LIVMARLI® (maralixibat) oral resolution is an oral, once-daily ileal bile acid transporter (IBAT) inhibitor accepted by the U.S. Meals and Drug Administration for the remedy of bile in sufferers with Alagille syndrome (ALGS) at three months of age Stasis pruritus and progressive familial intrahepatic cholestasis (PFIC) in 5 years of age and older.
LIVMARLI can be the one IBAT inhibitor accepted by the European Fee for the remedy of cholestatic pruritus in sufferers with ALGS 2 months and older and Well being Canada accepted for the remedy of cholestatic pruritus in ALGS. For extra data for U.S. residents, please go to LIVMARLI.com.
Mirum has additionally submitted LIVMARLI for European approval for the remedy of PFIC in sufferers two months of age and older.
LIVMARLI has acquired ALGS and PFIC Sort 2 Breakthrough Remedy Designations and ALGS and PFIC Orphan Drug Designations. To study extra about LIVMARLI’s ongoing scientific trials, please go to the Scientific Trials part of Mirum’s web site.
Vital security data
Utilization restrictions: LIVMARLI shouldn’t be appropriate for sufferers with PFIC sort 2 who’ve extreme defects within the bile salt export pump (BSEP) protein.
LIVMARLI could trigger negative effects, together with:
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liver harm. Sure liver take a look at modifications are widespread in individuals with Alagille syndrome and PFIC however could worsen throughout remedy. These modifications could also be indicators of liver harm. In PFIC, this may be severe or could result in liver transplantation or loss of life. Your healthcare supplier ought to do blood checks and a bodily examination to examine your liver operate earlier than beginning and through remedy. Inform your healthcare supplier straight away when you’ve got any indicators or signs of liver issues, together with nausea or vomiting, yellowing of the pores and skin or white components of the eyes, darkish or brown urine, ache on the fitting aspect of the abdomen (stomach), abdomen Swelling of the stomach, lack of urge for food, and bleeding or bruising extra simply than standard.
Abdomen and intestinal (gastrointestinal) issues. LIVMARLI may cause abdomen and intestinal issues, together with diarrhea and abdomen ache. Your healthcare supplier could advocate that you just monitor for brand spanking new or worsening abdomen issues, together with abdomen ache, diarrhea, bloody stools, or vomiting. When you expertise these signs extra typically or extra severely than standard, inform your well being care supplier straight away.
a situation known as Fats-soluble vitamin (FSV) deficiency This symptom, attributable to low ranges of sure nutritional vitamins (nutritional vitamins A, D, E and Ok) saved in physique fats, is widespread in individuals with Alagille syndrome and PFIC however could worsen throughout remedy. Your healthcare supplier ought to carry out blood checks earlier than beginning remedy and through remedy and should monitor for fractures and bleeding, which have been reported as widespread negative effects.
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US Prescription Info
EUSmPC
Canadian product monograph
About Miram Prescription drugs
Mirum Prescription drugs, Inc. is a biopharmaceutical firm devoted to reworking the remedy of uncommon illnesses affecting kids and adults. Mirum has three accepted medicines: LIVMARLI ® (maralixibat) oral resolution, CHOLBAM ® (cholic acid) capsules and CHENODAL ® (chenodiol) tablets.
LIVMARLI is an IBAT inhibitor accepted to deal with two uncommon liver illnesses that have an effect on kids and adults. It’s accepted in america (three months and older), Europe (two months and older), and elsewhere globally for the remedy of cholestatic pruritus in sufferers with Alagille syndrome. It’s also accepted in america for the remedy of cholestatic pruritus in sufferers 5 years and older with PFIC. Mirum has utilized for approval in Europe as a PFIC for the remedy of sufferers two months of age and older. CHOLBAM is FDA-approved for the remedy of bile acid synthesis problems attributable to a single enzyme deficiency and as an adjunctive remedy of peroxisomal problems in sufferers with indicators or signs or liver illness. CHENODAL has acquired approval of medical necessity from the FDA for the remedy of sufferers with cerebrotendinous xanthomatosis (CTX).
Mirum’s late-stage growth pipeline contains two investigational therapies for debilitating liver illnesses. Volixibat is an IBAT inhibitor being evaluated in two potential registration research, together with the Part 2b VISTAS examine in major sclerosing cholangitis and the Part 2b VANTAGE examine in major biliary cholangitis. Lastly, CHENODAL has been evaluated in RESTORE, a Part 3 scientific examine, for the remedy of sufferers with CTX, with constructive topline outcomes reported in 2023.
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To study extra about Mirum, go to mirumpharma.com and comply with Mirum on Fb (NASDAQ: ), LinkedIn, Instagram and Fb Twitter(X).
forward-looking statements
this The press launch accommodates forward-looking statements associated to the Firm’s plans to take part in scientific conferences, together with knowledge presentation titles and summaries, which can embody a dialogue of the Firm’s scientific and analysis knowledge associated to the therapeutic potential and/or business viability of LIVMARLI in liver illness indications and Examine-only affected person inhabitants. As a result of such statements are topic to dangers and uncertainties, precise outcomes could differ materially from these expressed or implied by such forward-looking statements. Phrases reminiscent of intentions, objectives, potential and related expressions are meant to determine forward-looking statements. The accuracy of such statements is topic to quite a few dangers, uncertainties and assumptions, together with, however not restricted to, the next: uncertainties inherent in analysis and growth; uncertainties inherent in enterprise and monetary planning, together with, however not restricted to, these associated to Mirum dangers associated to our enterprise and prospects, adversarial developments in our focus markets, or adversarial developments within the U.S. or international regulatory atmosphere or economies; the continuing impression of COVID-19 on our enterprise, operations and monetary efficiency; and aggressive developments. Different components that might trigger such variations embody these mentioned within the corporations’ filings with the SEC. All forward-looking statements contained on this press launch communicate solely as of the date hereon and are primarily based on administration’s assumptions and estimates as of that date. Mirum undertakes no obligation to replace such statements to replicate occasions that happen or circumstances that exist after the date the statements had been made, besides as required by regulation.
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View supply model on businesswire.com: https://www.businesswire.com/information/dwelling/20240518739027/en/
Media contact data:
Erin Murphy
media@mirumpharma.com
Investor contact data:
Andrew McKibben
traders@mirumphama.com
Supply: Miracle Prescription drugs, Inc.
