FDA Approves Ipsen’s Bylvay for Patients Living with Cholestatic Pruritus Due to Alagille Syndrome

Rare Daily Staff

The U.S. Food and Drug Administration has approved Ipsen’s Bylvay for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome.

Alagille syndrome (ALGS) is an inherited rare, genetic disorder that can affect multiple organ systems in the body including the liver, heart, skeleton, eyes and kidneys. Liver damage may result from having fewer than normal, narrowed or malformed bile ducts, which leads to toxic bile acid build-up, which in turn can cause scarring and progressive liver disease. Approximately 95 percent of patients with the condition present with chronic cholestasis, usually within the first few months of life and as many as 88 percent also present with severe, intractable pruritus.2,3 The estimated global incidence of ALGS is 3 in 100,000 live births. Currently in the United States, it is estimated that there are 1,300 patients who may be eligible for treatment with Bylavy.

Bylvay is a once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) that acts locally in the small intestine and has minimal systemic exposure. Bylvay was approved as the first drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC) in the United States, and for the treatment of PFIC in Europe, in 2021. Bylvay is immediately available via prescription for eligible ALGS patients.

“Today’s approval of Bylvay in a second indication allows patients and physicians to access an additional treatment option that has the potential to improve the management of pruritus, or intense itch, in this distressing condition that tends to affect young children,” said Howard Mayer, executive vice president and head of Research and Development for Ipsen.

Positive data from the phase 3 ASSERT study demonstrated that Bylvay provided highly statistically significant and clinically meaningful sustained improvements in pruritus, starting early after initiation of treatment. More than 90 percent of patients were pruritus responders. The overall incidence of treatment-emergent adverse events was similar to placebo. No patients discontinued the study and 96 percent of patients rolled over into the open-label extension study.

Ipsen has also submitted Bylvay to the European Medicines Agency, seeking authorization for ALGS, with Committee for Medicinal Products for Human Use opinion expected in the second quarter of 2023 and final EMA regulatory decision anticipated in second half of 2023. Bylvay has received orphan exclusivity for the treatment of PFIC, and Orphan Drug designations for the treatment of ALGS and biliary atresia, in the United States and Europe.

Bylvay is already FDA-approved for the treatment of pruritus in patients aged three months and older with all types of PFIC, and in Europe for the treatment of all types of PFIC in patients aged six months or older. In a third indication, the rare pediatric cholestatic liver disease, biliary atresia. Bylvay is in late-stage development with the phase 3 BOLD trial.

Photo: Howard Mayer, executive vice president and head of Research and Development for Ipsen