After Ipsen made a splash at this year's J.P. Morgan Healthcare Conference by scooping up liver disease specialist Albireo Pharma, the centerpiece of the buyout, Bylvay, has won a coveted label expansion.
First approved by the FDA to treat cholestatic pruritus from progressive familial intrahepatic cholestasis, Bylvay has now won an FDA approval in another liver disease. Specifically, the drug can now treat cholestatic pruritus due to Alagille syndrome in patients 12 months of age and older. Cholestatic pruritus is the intense itching that afflicts patients with the diseases.
A once-daily ileal bile acid transport inhibitor (IBATi), Bylvay will challenge Mirum Pharmaceuticals' Livmarli in this disease. Livmarli won an FDA approval in Allagile syndrome back in 2021 and generated $29 million during the first quarter of 2023. Analysts with Evercore ISI have previously put a $1 billion peak sales target on Mirum's rival drug.
Bylvay's new approval is based on results from the phase 3 ASSERT study, which showed that the drug provided "highly statistically significant and clinically meaningful sustained improvements in pruritus," according to Ipsen's release Tuesday.
In the study, more than 90% of patients who received the drug experienced at least some pruritus relief, according to the company.
With the Alagille syndrome nod, Ipsen plans an "immediate" commercial launch among eligible patients in the U.S. In Europe, the company is expecting a decision from regulators in the second half of the year.
Ipsen picked up the drug in its January acquisition of Albireo. Ipsen revealed that deal on the opening day of the industry's biggest annual conference, JPM.
Aside from its two approved uses, Bylvay is also in late-stage development for the rare pediatric cholestatic liver disease biliary atresia, Ipsen said.
"We are proud to have achieved FDA approval for Bylvay as a treatment for ALGS in the U.S. and we are committed to making it available to many more eligible patients across the world," Ipsen's R&D head Howard Mayer, M.D., said in a statement.
Ipsen, helmed by former Sanofi Pasteur chief David Loew, is on a mission to replace falling revenues from acromegaly drug Somatuline. Also at the JPM conference, Loew told Fierce Pharma about his plans to focus on certain rare diseases but stay away from the gene therapy platform for now.
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