New Therapy for Alagille Syndrome Estimated at $250,000 for an Annual Regimen

On September 29, the FDA approved the first medication for Children with Alagille syndrome (ALGS), Livmarli, a minimally absorbed ileal bile acid transporter (iBAT) inhibitor. (1)

Alagille is a rare genetic syndrome that mostly affects the liver and other organs of the body. Patients with ALGS have fewer small bile ducts than normal, resulting in bile build up in the live. This leads to liver damage and scarring. The disease usually starts in childhood, and can be manifested through a variety of symptoms such as itchy skin and malnutrition. (2)

While pricing has not been disclosed yet, Evercore estimated that Livmarli may have a price tag of about $250,000 for an annual regimen. Other therapies in this category, with a similar iBAT inhibitor mechanism of action, such as Bylvay, have a higher price tag around $385,000 yearly. (1)

The approval and availability for Livmarli, presents much needed treatment options for patients with ALGS.  For sponsors this is yet another example of complex therapies coming to market for a smaller subset of the population, with complicated management requirements. Plan sponsors must take a proactive approach to managing these drugs for their population across pharmacy and medical benefit. If plan sponsors fail to have robust strategies, there could be serious consequences to their bottom line.