A new tablet formulation of maralixibat could offer an alternative to the oral solution, with once-daily dosing among patients with Alagille syndrome and twice-daily dosing among those with progressive familial intrahepatic cholestasis.

The formulation is intended to treat those who may benefit from a solid-dose option. According to a press release from Mirum Pharmaceuticals, the U.S. Food and Drug Administration has approved the formulation for the treatment of cholestatic pruritus in these patient populations.

Maralixibat is an ileal bile acid transporter inhibitor approved in the United States for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 3 months and older and in those with progressive familial intrahepatic cholestasis (PFIC) aged 12 months and older. In Europe, it is approved for ALGS in patients 2 months and older and for PFIC in patients 3 months and older. The drug is currently under investigation in the phase III EXPAND trial for potential use in additional cholestatic conditions.

Liver-related adverse events, including elevated liver enzymes and hepatic injury, have been reported and may be severe in patients with PFIC. The prescribing information recommends baseline and ongoing monitoring of liver function tests.

Maralixibat is not indicated for PFIC type 2 in patients with severe defects in the bile salt export pump protein. Additional adverse effects include gastrointestinal symptoms such as diarrhea and abdominal pain as well as worsening fat-soluble vitamin (FSV) deficiencies, which are common in the target populations. Monitoring for FSV levels, bone health, and bleeding risk is advised during treatment.

Maralixibat should be administered orally 30 minutes prior to a meal. Mirum Pharmaceuticals anticipates tablet availability beginning in June through its Mirum Access Plus program.