FDA Expands Approval of Mirum’s Livmarli for Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

Image Credit: Adobe Stock Images/Olivier Le Moal

Mirum Pharmaceuticals announced that the FDA has approved Livmarli (maralixibat) for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). Additionally, the company has submitted a supplemental New Drug Application (sNDA) to start a higher concentration formulation of the treatment for label expansion to younger patients. The approval was based on data from the Phase III MARCH study, which demonstrated the drug's effectiveness across various genetic subtypes of PFIC.¹

“PFIC is a difficult disease for both the patient and family and significantly impedes the quality of life for all,” said Emily Ventura, executive director, PFIC Network in a press release. “These young and fragile patients endure an itch so severe that they experience deficits in their sleep, nutrition, growth, and, in the past, some have turned to transplant to resolve their itch. We are hopeful that Livmarli will have a measurable impact for patients, potentially offering a new normal and relief from the sleepless nights and disruption to their lives caused by cholestatic pruritus.”

The study, which included a total of 93 patients with multiple types of genetic PFIC, including PFIC1, PFIC2, PFIC3, PFIC4, and PFIC6, was reportedly the largest randomized trial conducted for the disease.¹

“Livmarli has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” said Chris Peetz, CEO, Mirum, in the press release. “Thank you to the patients, families, and clinicians whose study participation made this approval possible.”

Livmarli already has approval for the treatment of cholestatic pruritus in Alagille syndrome (ALGS)patients in the United States, Canada, Europe, and other regions.¹

“Livmarli’s approval in cholestatic pruritus for patients with PFIC is a result of years of investigation and a collection of a strong body of clinical evidence showing meaningful improvements across a number of important parameters, including pruritus, affecting children with PFIC,” said Richard Thompson, professor of molecular hepatology, King’s College London, MARCH study investigator, in the press release. “I am pleased that we will have a well-studied and efficacious option to offer patients whose life has been disrupted by itch.”

Livmarli was first approved by the FDA in 2021 for the treatment of ALGS in patients over the age of 1 year. This approval was based on positive data from Mirum’s ICONIC study in combination with five years of previous data on 86 patients with ALGS. The study showed significant reductions in pruritus, one of the most common symptoms associated with the disease.²

“We have had the pleasure of being part of and closely following the clinical progress of Livmarli in many ways. Since the first study’s initiation more than a decade ago, we have dreamed of today, seeing Livmarli receive FDA approval, marking an incredibly meaningful milestone for the ALGS community,” said Roberta Smith, president, Alagille Syndrome Alliance, in the press release. “Until now, patients have had limited-to-no treatment options to address the severe and unrelenting itch that significantly impacts both patients and their families. Additionally, because pruritus associated with ALGS greatly impacts caregivers, having a strong support program like Mirum Access Plus to reduce the strain on families is so important. The ALGS community has been waiting for a long time for a treatment and we’re so pleased that Livmarli is now available in the United States.”

References

1. Mirum Pharmaceuticals’ LIVMARLI Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis. Business Wire. March 13, 2024. Accessed March 14, 2024. https://www.businesswire.com/news/home/20240313135184/en/Mirum-Pharmaceuticals%E2%80%99-LIVMARLI-Receives-FDA-Approval-for-Treatment-of-Cholestatic-Pruritus-in-Patients-with-Progressive-Familial-Intrahepatic-Cholestasis

2. U.S. FDA Approves LIVMARLI (maralixibat) as the First and Only Approved Medication for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome One Year of Age and Older. Mirum. September 29, 2021. Accessed March 14, 2024. https://ir.mirumpharma.com/news-events/News/news-details/2021/U.S.-FDA-Approves-LIVMARLI-maralixibat-as-the-First-and-Only-Approved-Medication-for-the-Treatment-of-Cholestatic-Pruritus-in-Patients-with-Alagille-Syndrome-One-Year-of-Age-and-Older/default.aspx