Ipsen (Euronext: IPN; ADR: IPSEY) presented new data at the American Association for the Study of Liver Diseases (AASLD) from two Phase III open-label extension studies, evaluating the long-term efficacy and safety of Bylvay® in patients with Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille syndrome (ALGS). The data revealed sustained efficacy with improvements in height, weight, and sleep for patients treated with Bylvay for at least 72 weeks in both conditions.
Sandra Silvestri, EVP Chief Medical Officer at Ipsen, emphasized the impact of diseases like PFIC and ALGS on patients and their families, noting that Bylvay’s sustained efficacy and favorable safety profile are vital for improving patient outcomes.
PEDFIC 2 Study in PFIC
The open-label extension of the PEDFIC 2 study (n=116) evaluated Bylvay’s efficacy and safety over 72 weeks. Results showed a 1-point reduction in pruritus score in 42% of patients under 18 with PFIC 1 and 2, and in 61% of all PFIC patients of any age, with rapid improvements in pruritus sustained through week 72. Serum bile acid (sBA) levels also decreased significantly, with a mean reduction of 104 µmol/L in patients who transitioned to Bylvay at week 24, and a reduction of 58 µmol/L in those who stayed on Bylvay. Improvements in height, weight, and sleep were also reported, with most adverse events being mild to moderate, such as gastrointestinal issues.
ASSERT-EXT Study in ALGS
The ASSERT-EXT study (n=50) showed sustained improvements in pruritus and sBA levels in ALGS patients treated with Bylvay for 72 weeks. A significant reduction in pruritus (≥1 point) was observed in 93% of patients who received continuous Bylvay and 77% of those who transitioned from placebo. The mean reduction in sBA was 124 µmol/L for those on continuous Bylvay and 139 µmol/L for those who switched from placebo. Additionally, patients showed improvements in height (8.2 cm) and weight (2.8 kg) with continuous Bylvay use, and 10.7 cm and 3.3 kg in those who switched from placebo. Sleep improvements were noted across all parameters. The safety profile was consistent with previous trials, with mild to moderate adverse events, primarily gastrointestinal, and one treatment discontinuation due to a side effect.
These findings underscore the potential of Bylvay to manage symptoms and improve quality of life for patients with PFIC and ALGS, while supporting its ongoing safety and tolerability.
