Mirum Pharmaceuticals Submits Supplemental New Drug Application for LIVMARLI in Patients with Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis
Mirum Pharmaceuticals has announced that it has submitted a supplemental New Drug Application (sNDA) for LIVMARLI (maralixibat) oral solution for the treatment of cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC).
The sNDA submission is based on data from the MARCH PFIC Phase 3 study of LIVMARLI. MARCH PFIC is the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. In the study, 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), in the cohort evaluating combined genetic subtypes. The sNDA also includes data from the Phase 2 INDIGO study of PFIC2 patients demonstrating transplant-free survival in all serum bile acid responders after more than five years of treatment with LIVMARLI.
LIVMARLI is currently approved in the U.S. and Israel for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, as well as in the European Union in the same indication, in patients two months of age and older.
“Following the groundbreaking MARCH clinical data, we are thrilled at the opportunity to provide the PFIC community in the U.S. a treatment option that significantly reduces pruritus and gives hope for a life less burdened by PFIC,” said Chris Peetz, president and chief executive officer at Mirum. “The MARCH PFIC study greatly advanced the understanding of treatment options for PFIC by including the broadest range of genetic types ever studied. We are working urgently to bring LIVMARLI to PFIC patients.”
“PFIC patients suffer greatly due to the unrelenting pruritus associated with the disease and the potential to have another treatment option available with such compelling data to support its use is really exciting,” said Emily Ventura, executive director, PFIC Network and mom to child with PFIC. “The results seen in the LIVMARLI clinical studies are encouraging as they help to show the potential benefit that patients treated with this medication may experience, which provides hope for a healthier tomorrow.”
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