US FDA approves expanded use of Mirum`s liver disease drug
Mirum’s LIVMARLI® Now Authorized in Canada for Cholestatic Pruritus in Patients with Alagille Syndrome
Mirum Pharmaceuticals` Livmarli (Maralixibat chloride) Approved In Europe
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the 55th European Society for Pediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting. Among the data presented were four oral and three poster presentations featuring analyses from LIVMARLI® (maralixibat) oral solution clinical studies.
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that Hepatology Communications published data from the CAMEO study evaluating the safety, tolerability, and efficacy of LIVMARLI® (maralixibat) oral solution in patients with primary sclerosing cholangitis (PSC).
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced submission to the European Medicines Agency (EMA) of a variation application to the Marketing Authorization for LIVMARLI® (maralixibat) oral solution, to extend the label for progressive familial intrahepatic cholestasis (PFIC) in patients two months of age and older. LIVMARLI is currently approved by the EMA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. nn“The expansive data we have collected from our PFIC studies give us great confidence that, if approved by the EMA, LIVMARLI will provide an impactful treatment option for patients across a broad range of genetic types, including those with phenotypic diagnosis but without a known variant” nnTweet this nThe submission is based on data from the Phase 3 MARCH study of LIVMARLI in patients with PFIC. MARCH is the largest randomized trial conducted in PFIC, with 93 patients across a broad range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. In the cohort evaluating combined genetic types, LIVMARLI-treated patients had statistically significant improvements in pruritus (p< 0.0001), serum bile acids (p<0.0001), bilirubin (p=0.0471), and growth as measured by weight z-score (p=0.0391). The variation also included interim analysis data from the ongoing open-label extension study (MARCH-ON) which included 85 of the 93 patients that were enrolled in the pivotal trial. Overall, LIVMARLI demonstrated maintenance of treatment effect with sustained reductions in serum bile acid and bilirubin levels as well as continuous pruritus improvement. Growth acceleration was further improved throughout the follow-up indicating catch-up growth in children with PFIC. nn“The expansive data we have collected from our PFIC studies give us great confidence that, if approved by the EMA, LIVMARLI will provide an impactful treatment option for patients across a broad range of genetic types, including those with phenotypic diagnosis but without a known variant,” said Chris Peetz, president and chief executive officer at Mirum. “We have now submitted these groundbreaking data to regulatory agencies in the U.S. and Europe with the hope of making LIVMARLI available to PFIC patients across multiple geographies. We are grateful to the clinicians and families who participated in the research that has meaningfully advanced treatment for PFIC.” nnLIVMARLI is currently approved in the United States and Europe for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and two months of age and older, respectively. In addition to the Marketing Authorization variation announced today, Mirum has also submitted a supplemental new drug application to the U.S. FDA for the treatment of cholestatic pruritus in patients three months of age and older with PFIC. Regulatory submissions outside of the U.S., Canada and Europe are being pursued by partners and distributors in key markets globally. nnAbout LIVMARLI® (maralixibat) oral solution nnLIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com. nnMirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe (in PFIC for patients two months of age and older). nnLIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. nnIMPORTANT SAFETY INFORMATION nnLIVMARLI can cause side effects, including: nnChanges in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests 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) or loss of appetite. nnStomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. nnA condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment. nnOther common side effects reported during treatment were gastrointestinal bleeding and bone fractures. nnUS Prescribing Information nnEU SmPC
Mirum Pharmaceuticals Submits Supplemental New Drug Application to FDA for LIVMARLI in Patients with Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis