Mirum Pharmaceuticals announces the validation by the European Medicines Agency of the marketing authorization application for maralixibat in patients with PFIC2
– The five-year transplant-free survival data from the phase 2 INDIGO study served as the basis for submitting the Marketing Authorization; .
– Maralixibat would be the first labeled treatment for patients with PFIC2 in Europe, if approved
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver disease, today announced that the Marketing Authorization Application (MAA) for the company for its investigational drug, maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as bile salt export pump deficiency (BSEP), was accepted for review (validated) by the European Medicines Agency (EMA). The validation of the application by the EMA confirms that all essential regulatory elements are included in the submission so that the EMA can begin their review.
“PFIC is changing the lives of patients and their families as they struggle to manage the 24-hour care and surgical decisions that many children often need,” said Chris Peetz, President and CEO of Mirum. “The validation of our MAA is a revolutionary step towards providing a drug to treat PFIC2. Based on the improvement in long-term transplant-free survival in maralixibat responders, we believe that maralixibat may provide a treatment alternative to invasive surgeries for these patients, as well as improve quality of life. We are excited about the opportunity to make maralixibat available to patients with PFIC2 in Europe. “
Data from the INDIGO phase 2 study evaluating maralixibat in pediatric patients with PFIC2 were used as the basis for submitting the Marketing Authorization. Mirum recently ad data showing five-year transplant-free survival for patients who achieved serum bile acid control. The data also showed improvements in several parameters, including control of pruritus, improvements in liver enzymes and bilirubin levels, and improved growth. These data were presented at the annual meeting of the European Association for the Study of the Liver. The MAA submission also includes data on five-year event-free survival with maralixibat compared to the NAPPED natural history cohort.
To provide further evidence for the potential of maralixibat in PFIC2 with higher doses and other PFIC subtypes, Mirum is conducting a phase 3 study, MARCH, with registrations expected to be completed in the second quarter of 2021.
In addition to the MA application for maralixibat in PFIC2, Mirum has also initiated a continuous new drug application (NDA) with the United States Food and Drug Administration (FDA) for maralixibat for the treatment of Cholestatic pruritus in patients with Alagille syndrome (ALGS). Mirum plans to complete the submission in the first quarter of 2021, with a launch slated for the second half of the same year. The company also recently launched an expanded access program making maralixibat available to eligible patients with ALGS in the United States, Canada, Australia and certain countries in Europe.
Maralixibat is a new investigational drug administered orally, poorly absorbed and evaluated in several cholestatic liver diseases. Maralixibat inhibits the apical sodium-dependent bile acid transporter (ASBT), which results in the excretion of more bile acids in the stool, resulting in a systemic drop in bile acid levels, reducing thus potentially bile acid-mediated liver damage and associated effects and complications. More than 1,600 people have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as a reduction in xanthomas and long-term accelerated growth. In one Phase 2 PFIC study, a genetically defined subset of deficient BSEP (PFIC2), patients responded to maralixibat. The United States Food and Drug Administration (FDA) has granted breakthrough therapy designation to maralixibat for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well tolerated throughout the studies. The most common treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved by regulatory authorities and available for prescription, the drug is available for patients with ALGS through Mirum’s Expanded Access Program. For more information, please visit ALGSEAP.com. For more information on the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.
About Mirum Pharmaceuticals
Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of an advanced-stage pipeline of novel therapies for debilitating liver disease. The Company’s flagship product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia. The Company has initiated a continuing NDA application for maralixibat in the treatment of cholestatic pruritus in patients with ALGS and expects to complete the submission in the first quarter of 2021. In addition, the application for authorization to put on the Mirum’s market for the treatment of pediatric patients with PFIC2 was accepted. for review (validated) by the European Medicines Agency.
Mirum is also developing volixibat, also an oral ASBT inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information visit MirumPharma.com.
Statements contained in this press release concerning matters which are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include statements concerning, among other things, results, conduct and progress of Mirum’s ongoing studies for maralixibat and the regulatory approval process for maralixibat. Since these statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by these forward-looking statements. Words such as “plans”, “will”, “may”, “expects”, “,, as well.” These forward-looking statements are based on Mirum’s current expectations and involve assumptions which may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in these forward-looking statements due to various risks and uncertainties, which include, without limitation, the risks and uncertainties associated with Mirum’s business in general, the impact of the COVID pandemic -19 and the other risks described in documents filed by Mirum with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of that date. Mirum assumes no obligation to update these statements to reflect events that occur or circumstances that exist after the date they were made, except as required by law.
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Ian Clements, Ph.D.