EC Approves Ipsen's Kayfanda for Alagille Syndrome Pruritus Treatment

Ipsen’s Kayfanda (odevixibat) has received approval from the European Commission (EC) under exceptional circumstances for the treatment of cholestatic pruritus in patients aged six months and older who have Alagille syndrome (ALGS). This syndrome is a rare genetic disorder that occurs in about three out of every 100,000 births globally. It can impact multiple organ systems, including the liver, heart, skeleton, eyes, and kidneys. Symptoms typically appear within the first three months of life and include bile flow blockage from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), and severe itching (pruritus).

Kayfanda is an oral medication that acts as a non-systemic bile acid transport inhibitor. Ipsen acquired access to this drug last year through its $952 million purchase of rare disease specialist Albireo. The drug is already approved in the European Union under the brand name Bylvay for the treatment of progressive familial intrahepatic cholestasis in patients aged six months and older.

The European Commission's recent decision to approve Kayfanda for ALGS was based on positive outcomes from the late-stage ASSERT trial. This trial demonstrated statistically significant and clinically meaningful improvements from baseline to six months in the severity of scratching for patients treated with Kayfanda compared to those given a placebo. Additionally, there was a statistically significant reduction in serum bile acid concentration at the end of the treatment period for patients on Kayfanda versus those on placebo. The incidence of treatment-emergent adverse events was similar between the two groups.

Christelle Huguet, executive vice president and head of research and development at Ipsen, commented on the approval. She highlighted the severe impact of pruritus on the quality of life for patients with ALGS, describing it as one of the most significant symptoms of the condition. Huguet expressed Ipsen's commitment to making this new treatment option available for use by patients in the European Union.

Just days before this approval, Ipsen’s Iqirvo (elafibranor) also received conditional approval from the European Commission for the treatment of primary biliary cholangitis, another rare cholestatic liver disease. This condition leads to irreversible fibrosis (scarring) of the liver and destruction of the bile ducts. Iqirvo is an oral peroxisome proliferator-activated receptor agonist and was approved for use in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA. It was also approved as a monotherapy for patients who cannot tolerate UDCA.

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