Terri Airov

All articles by Terri Airov

FDA Approves First Treatment for Cholestatic Pruritus in Patients With Alagille Syndrome

The US Food and Drug Administration (FDA) has approved maralixibat (Livmarli™) as the first treatment for cholestatic pruritus in patients with Alagille syndrome (ALGS) who are aged 1 year or older, Mirum Pharmaceuticals, Inc. announced. It is now available for prescribing. Maralixibat, which is administered orally once-daily, is a minimally absorbed ileal bile acid transporter…


FDA Approves New Enzyme Replacement Therapy for Late-Onset Pompe Disease

The US Food and Drug Administration (FDA) has approved Sanofi Genzyme’s avalglucosidase alfa-ngpt (Nexviazyme®), an enzyme replacement therapy (ERT), for the treatment of late-onset Pompe disease (LOPD) in patients who are at least 1 year old. Avalglucosidase alfa helps reduce the accumulation of glycogen that occurs in the skeletal and heart muscles of patients with…

Next post in Pompe News Briefs