In February 2023, the FDA accepted an sNDA submitted by Albireo, seeking to add the rare genetic disease Alagille syndrome to Bylvay (odevixibat)’s label. The target action date is June 15. Albireo entered into an agreement in January to be acquired by Ipsen
Alagille syndrome is a heritable condition characterized by the lack of bile ducts that drain the liver, leading to the accumulation of bile, which ultimately causes organ damage. The disease can also affect the heart, skeleton, eyes, kidneys and central nervous system. Common symptoms include yellow skin or eyes, stunted growth and severe pruritus.
Bylvay’s Alagille bid is supported by data from the Phase III ASSERT study, a double-blinded, randomized and placebo-controlled trial that enrolled more than 50 participants across 32 trial sites in North America, the Middle East, Europe and the Asia-Pacific region.
In the study’s main analysis, Bylvay significantly reduced pruritus after 6 months of treatment as compared with placebo, thereby meeting ASSERT’s primary endpoint. The drug also significantly lowered serum bile acid levels and improved sleep parameters, according to Albireo.
As for safety, ASSERT found Bylvay to be well-tolerated, with a side effect profile that was comparable to placebo. None of the patients dropped out due to toxicities.
The FDA also granted Bylvay’s sNDA its Priority Review designation.