Medison and Ipsen’s Bylvay
Health Canada approves Medison and Ipsen’s Bylvay to treat ALGS
Health Canada has approved Medison Pharma and Ipsen’s Bylvay (odevixibat) to treat cholestatic pruritus in patients aged 12 months and above with Alagille syndrome (ALGS). Alagille syndrome can impact the liver, heart and kidneys. The approval is backed by the placebo-controlled, randomised and double-blind Phase III ASSERT trial.
This follows Bylvay’s initial Canadian approval in 2023 for pruritus in patients six months and older with progressive familial intrahepatic cholestasis (PFIC), a severe liver condition.
ALGS is a rare genetic disorder impacting organs including the liver, heart and kidneys, and affects 1 in 30,000 live births. The approval by Health Canada was based on results from the global Phase III ASSERT study, which confirmed Bylvay’s effectiveness in alleviating cholestatic pruritus in ALGS patients within weeks of treatment.
ASSERT is the first and only Phase III trial completed for ALGS patients. The study also reported secondary benefits, such as improved sleep quality and reduced bile acid levels, alongside a favourable safety profile.
Medison and Ipsen collaborate in Canada and Israel to provide Bylvay to patients with these rare diseases.
Bylvay, a non-systemic ileal bile acid transport inhibitor (IBATi), is administered once daily and targets the small intestine with minimal systemic exposure. It addresses key challenges for ALGS patients by reducing itching and lowering bile acid levels. Bylvay offers flexible dosing based on body weight and is available in capsule form, which can be swallowed or mixed with food or liquids.
Initially launched in the US in 2021 for PFIC treatment, Bylvay received approval in the EU the same year.In June 2023, Bylvay was approved in the US for treating cholestatic pruritus in ALGS patients aged 12 months and older.
The ASSERT trial, a double-blind, randomised, placebo-controlled study, assessed the safety and efficacy of 120 µg/kg/day odevixibat over 24 weeks in ALGS patients. Conducted across 32 sites in Asia Pacific, Europe, North America and the Middle East, the trial included individuals from birth to 17 years with genetically confirmed ALGS.
The primary analysis showed a significant improvement in pruritus, as measured by the PRUCISION Observer-Reported Outcome scratching score, compared to the placebo group.
The study also achieved a key secondary endpoint, demonstrating a significant decrease in serum bile acid concentration. Odevixibat treatment further improved sleep parameters, including the percentage of days with assistance in falling asleep and reduced daytime tiredness.
