FDA approves Bylvay for cholestatic pruritus due to Alagille syndrome
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The FDA has approved Bylvay for the treatment of cholestatic pruritus due to Alagille syndrome in patients aged 12 months or older, according to an Ipsen Biopharmaceuticals press release.
This approval marks the second rare cholestatic liver disease indication for Bylvay (odevixibat, Ipsen/Albiero Pharmaceuticals), a non-systemic ileal bile acid transport inhibitor originally approved in 2021 for pruritus in all subtypes of progressive familial intrahepatic cholestasis.
“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,” Howard Mayer, executive vice president and head of research and development for Ipsen, said in the release. “We are proud to have achieved FDA approval for Bylvay as a treatment for [Alagille syndrome (ALGS)] in the U.S. and we are committed to making it available to many more eligible patients across the world.”
The FDA based their decision on results from the ASSERT phase 3 study, a double-blind, randomized, placebo-controlled trial which assessed the safety and efficacy of odevixibat 120 µg /kg/day for 24 weeks for relieving pruritus in patients with ALGS from 32 sites across North America, Europe, Middle East and the Asian Pacific. The study enrolled patients aged 0 to 17 years old with a genetically confirmed diagnosis of ALGS.
According to study results, odevixibat provided statistically significant and clinically meaningful sustained improvement of pruritis as early as initiation of treatment. Additionally, more than 90% of patients who received odevixibat were pruritus responders. Study results also demonstrated a highly statistically significant reduction in serum bile acid concentration from baseline to the average of weeks 20 and 24 vs. placebo (P=.001). Statistically significant improvements were also reported for sleep parameters as early as weeks 1-4.
Compared with placebo, odevixibat had a similar overall incidence of treatment-emergent adverse events, with a low incidence of drug-induced diarrhea. No patients were reported to have discontinued treatment, with 96% of patients continuing into the open-label extension study. The most common adverse events associated with odevixibat were diarrhea, abdominal pain, hematoma and weight loss.
“Physicians urgently need more options to treat patients with Alagille syndrome and this approval from the U.S. FDA spotlights the robustness of the phase 3 ASSERT clinical study results,” Nadia Ovchinsky, MD, chief of the division of gastroenterology and hepatology, Hassenfeld Children’s Hospital at NYU Langone, said in the release. “The ASSERT study showed that Bylvay reduced pruritus associated with ALGS, which is so common among this patient population and one of the leading indications for a liver transplant.”
The company noted that odevixibat is also in late-stage development with the BOLD phase 3 trial, working towards its third indication for biliary atresia.
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