Ipsen has received the European Commission’s (EC) approval for Kayfanda (odevixibat) to treat cholestatic pruritus in Alagille Syndrome (ALGS) in patients aged six months and older.
EC has approved this once-daily, non-systemic ileal bile acid transport (IBAT) inhibitor under exceptional circumstances.
Kayfanda, known as Bylvay outside the European Union (EU), was approved in the EU as the first drug option for all types of progressive familial intrahepatic cholestasis (PFIC) in patients aged six months and older.
In the US, it was approved in June last year for cholestatic pruritus treatment in patients from 12 months of age.
The EC approval for cholestatic pruritus in ALGS is based on the data from the double-blind, randomised, placebo-controlled Phase 3 ASSERT clinical trial. The study enrolled patients aged zero to 17 years with a genetically confirmed diagnosis of ALGS.
In the primary analysis, the study met its primary endpoint, showing a highly statistically significant improvement in pruritus.
This was measured using the PRUCISION Observer-Reported Outcome scratching score at month six, compared to the placebo group.
In addition, the study achieved a key secondary endpoint, demonstrating a significant reduction in serum bile acid concentration from baseline to the average at weeks 20 and 24.
Patients also reported improvements in multiple observer-reported sleep parameters. The overall incidence of treatment-related adverse events was similar to placebo.
Ipsen research and development head and executive vice president Christelle Huguet said: “Patients living with Alagille syndrome often endure a very poor quality of life as a result of the intolerable itch, which is one of the most significant symptoms of this condition.
“Today’s decision is therefore very welcome. We will now continue in our ongoing efforts to make this new treatment option available for use with patients living in the EU.”
Currently, Kayfanda is being studied in the Phase 3 BOLD trial for biliary atresia, whose data is expected in 2026.
Recently, Ipsen secured EU approval for Iqirvo (elafibranor) in combination with ursodeoxycholic acid (UDCA) to treat primary biliary cholangitis, enhancing its portfolio in rare cholestatic liver diseases.
