Ipsen’s Kayfanda (odevixibat) has been approved by the European Commission (EC) under exceptional circumstances to treat cholestatic pruritus in Alagille syndrome (ALGS) patients aged six months and older.
Occurring in approximately three in every 100,000 births globally, ALGS is a rare genetic disorder that can affect multiple organ systems in the body, including the liver, heart, skeleton, eyes and kidneys.
Symptoms usually develop during the first three months of life and include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), and severe itching (pruritis).
Kayfanda is an oral, non-systemic bile acid transport inhibitor that Ipsen gained access to after it acquired rare disease specialist Albireo last year for $952m.
The drug is already approved in the EU under the brand name Bylvay to treat progressive familial intrahepatic cholestasis in patients aged six months and older.
The EC’s latest decision was supported by positive results from the late-stage ASSERT trial, which demonstrated statistically significant and clinically meaningful improvements from baseline to six months in scratching severity for patients receiving Kayfanda versus those randomised to receive placebo.
A statistically significant reduction in serum bile acid concentration at the end of treatment was also demonstrated for patients on Kayfanda versus placebo, and the overall incidence of treatment emergent adverse events was similar across the two cohorts.
Christelle Huguet, executive vice president and head of research and development at Ipsen, said: “Patients living with ALGS 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… We will now continue in our ongoing efforts to make this new treatment option available for use with patients living in the EU.”
The approval came just days after Ipsen’s Iqirvo (elafibranor) received conditional approval from the EC to treat primary biliary cholangitis, a rare cholestatic liver disease that causes irreversible fibrosis (scarring) of the liver and destruction of the bile ducts.
The oral peroxisome proliferator-activated receptor agonist was specifically authorised for use in combination ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as a monotherapy in patients who are unable to tolerate UDCA.