News Release Details
Mirum Pharmaceuticals Announces Completion of Rolling NDA Submission for Maralixibat in Alagille Syndrome
- Maralixibat
The NDA submission included the results of the ICONIC study and additional supportive studies totaling 86 patients with ALGS with up to six-year follow-up. The clinical safety data submitted for maralixibat includes over 1,600 individuals treated.
“Patients, families and physicians have an urgent need for effective medications to address the severe and life-altering symptoms associated with ALGS,” said
Until maralixibat is approved by the FDA for prescription, maralixibat is available to eligible patients with ALGS through Mirum’s Expanded Access Program (EAP). The EAP is available in
Maralixibat was granted Rare Pediatric Disease Designation for ALGS and, as such, may qualify for receipt of a priority review voucher, if approved by the FDA. Maralixibat was also granted Breakthrough Therapy Designation for the treatment of pruritus associated with ALGS in patients one year of age and older. Maralixibat was granted Orphan Drug Designation by the FDA for the treatment of patients with PFIC and ALGS.
About Alagille syndrome
Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people.1 In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system.2 The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60% to 75% of patients with ALGS have a liver transplant before reaching adulthood.3 Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin), and pruritus (itch)2. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.4
About Maralixibat
Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals 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 reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The
About Mirum
Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.
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Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the regulatory approval path for maralixibat in
References
1Danks, et al. Archives of Disease in Childhood 1977
2
3Vandriel, et al. GALA EASL 2020; Kamath, et al.
4Elisofon, et al.
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