Ipsen’s drug Bvlay has gotten its second approval for itching (pruritis) in a rare liver disease, this time in Alagille syndrome. The drug had previously been approved for progressive familial intrahepatic cholestasis. Another indication may be on the way in the near future, as Ipsen is currently in late-stage testing for the drug in a childhood liver disorder.
According to Phil Taylor, “Alagille syndrome is caused by a mutation in the JAG1 gene, with symptoms generally appearing in the first two years of life. It causes liver damage, leading to jaundice and pruritus, as well as other effects including delayed growth and development, skin abnormalities, and cardiovascular complications. Itching is widely acknowledged to be one of the most debilitating symptoms of the disease, particularly in young children, and is one of the main drivers for patients to seek a liver transplant.”
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(Source: PharmaPhorum, June 14th, 2023)