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CHMP opinion based on pivotal ICONIC study with more than five years of data demonstrating durable and clinically meaningful improvements in cholestatic pruritus and serum bile acids for patients with Alagille syndrome (ALGS).
Lead Product(s): Maralixibat
Therapeutic Area: Genetic Disease Product Name: Livmarli
Highest Development Status: Approved Product Type: Small molecule
Recipient: Mirum Pharmaceuticals
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 14, 2022