Alagille Syndrome
Conference Roundup

Maralixibat proves effective for young adults with Alagille syndrome transitioning to adult care

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Maralixibat (MRX) has proven to be an effective, durable, and well-tolerated treatment for individuals aged 16 years and older with Alagille syndrome (ALGS), according to data presented at NASPGHAN 2023.

This study, which included both young adults transitioning to adult care and adults initiating treatment, demonstrated significant improvements in pruritus (itching) and serum bile acid levels. These positive results provide critical data supporting the continued use of MRX in adults with ALGS who have survived with their native livers into adulthood.

The study included 14 individuals, with 11 starting MRX treatment before the age of 16. There was a median age of 13 years at the onset of therapy. These patients underwent treatment for an average duration of 3.7 years, with the eldest recipient receiving MRX at 21 years. An additional 3 patients began MRX at or above 16 years of age and were monitored for an average of 3.1 years.

All patients received MRX doses tailored to their weight, with an adult maximum dose of 28.5mg per day. For those initiating MRX before the age of 16, pruritus levels significantly decreased from a baseline mean of 2.5 to 0.8 on the ItchRO[Obs] scale, signifying minimal to no itch (delta = -1.7; P = 0.002) prior to the age of 16. This improvement was sustained, with no substantial change observed before and after this critical age milestone (delta = -0.2; P = 0.2), or throughout the course of therapy (delta = 0.2; P = 3).

Baseline serum bile acid levels were 130 µmol/L, showing a significant reduction to 52 µmol/L (delta = -79; P = 0.03) before the age of 16. This decrease remained stable, with no significant alteration noted before and after age 16 (delta = -7; P = 0.3) or at the conclusion of therapy (delta = 3; P = 0.4).

For the 3 individuals initiating MRX at or above 16 years, pruritus also exhibited marked improvement from baseline, with delta values in ItchRO[Obs] ranging from -0.6 to -2.8. Although 1 patient experienced a substantial decrease in serum bile acids (delta = -112 µmol/L), 2 individuals showed slight increases (delta = 8 and 11 µmol/L).

MRX demonstrated consistent safety and tolerability profiles.

Hirschfield G, et al. Impact of Maralixibat on cholestatic pruritus in young adults aged 16 years and older with Alagille syndrome. Presented at: NASPGHAN 2023.

Rare Disease 360® is the Official Media Partner and Official Publication of The Alagille Syndrome Alliance (Alagille.org).