EU Backs Drug to Relieve Itching in Alagille Syndrome

A drug to treat severe itching in patients with Alagille syndrome should be granted a marketing authorization in the European Union under what the European Medicines Agency (EMA) termed exceptional circumstances.

At its monthly meeting, the agency’s Committee for Medicinal Products for Human Use (CHMP) determined that Kayfanda (odevixibat; Ipsen Pharma) is clinically effective for cholestatic pruritus and associated sleep disturbances in patients with Alagille syndrome. 

Alagille syndrome is a rare genetic disorder associated with abnormalities of the liver, heart, eyes, kidneys, and skeleton. Most people with the condition have mutations in one copy of the JAG1 gene. 

Early symptoms of Alagille syndrome can include cholestasis, jaundice, and pruritus.

Reducing the Concentration of Bile Acids

The active substance of Kayfanda is odevixibat, a reversible and selective inhibitor of the ileal bile acid transporter that acts locally in the distal ileum. It reduces bile acid reuptake and increases their clearance through the colon.

The CHMP endorsed available evidence suggesting that Kayfanda reduces the concentration of serum bile acids in patients with Alagille syndrome, easing pruritus and thereby improving sleep quality.

The most common side effects are diarrhea, abdominal pain, vomiting, and increases in hepatic enzymes.

The full indication for authorizing Kayfanda is the treatment of cholestatic pruritus in patients with Alagille syndrome aged 6 months or older.

Kayfanda will be available as 200-, 400-, 600-, and 1200-µg hard capsules.

Exceptional Circumstances

In exceptional circumstances, the EMA may recommend granting an authorization subject to certain specific obligations, which need to be reviewed each year. This happens when the applicant can show that they are unable to provide full data on the efficacy and safety of the medicinal product due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in collecting the data.

Granting of the marketing authorization is subject to ratification by the European Commission.