Mirum Pharmaceuticals to Present LIVMARLI® Data at NASPGHAN Annual Meeting 2024

Mirum Pharmaceuticals to Showcase LIVMARLI® Data at NASPGHAN Annual Meeting

Mirum Pharmaceuticals, Inc. is set to present pivotal clinical data on its FDA-approved treatment LIVMARLI® (maralixibat) during the upcoming NASPGHAN annual meeting scheduled for November 6-9, 2024, in Hollywood, Florida. This event serves as a significant platform for the company to share findings from its research aimed at treating pediatric patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). The presentations will cover a range of studies that highlight the efficacy and sustained impact of maralixibat on these complex liver diseases, reinforcing its position as a leading therapeutic option in the pediatric cholestatic liver disease landscape.

Among the key presentations is Abstract 77, which reports on growth improvements among PFIC patients stemming from the MARCH/MARCH-ON trials. These studies indicate that patients receiving LIVMARLI show considerable positive changes in growth metrics, underscoring the treatment's potential impact not only on liver function but also on overall health and development in affected children. Another significant contribution will be Abstract 361, which discusses long-term clinical benefits for ALGS patients, showcasing data that spans seven years from the MERGE study. This longevity of effect emphasizes the importance of continuous treatment and its implications for managing chronic conditions in young patients.

In addition to these studies, other abstracts will explore various aspects of maralixibat's therapeutic benefits. Abstract 637 will present findings on improved liver health markers in children with PFIC, while Abstract 638 will detail the correlation between reduced pruritus and enhanced quality of life for individuals with cholestatic liver disease. Furthermore, Abstract 664 will provide real-world data on the long-term impacts of maralixibat on concomitant medication use for managing cholestatic pruritus in children with ALGS. These findings collectively reinforce the significance of LIVMARLI as an effective treatment option that not only alleviates symptoms but also promotes better health outcomes in pediatric patients.

Mirum Pharmaceuticals continues to advance its commitment to addressing unmet medical needs in pediatric liver diseases. LIVMARLI, designed to inhibit the ileal bile acid transporter (IBAT), is already recognized for its efficacy in managing cholestatic pruritus in young patients, particularly those aged three months and older with ALGS and 12 months and older with PFIC in the United States. The upcoming NASPGHAN meeting presents an opportunity for Mirum to further engage with the medical community and share the promising results of its ongoing research.

As Mirum Pharmaceuticals prepares for its participation in this key industry event, it remains dedicated to improving the lives of children suffering from rare liver diseases. For more information on LIVMARLI, interested parties can visit LIVMARLI.com.