Ipsen’s Current Rare Disease Therapies: Approved and In Development +... ++++

https://checkrare.com/ FDA NEWS 2025 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs.  FDA Extends Evinacumab Indication to Include Patients With HoFH Ages 1 to 5 Years https://checkrare.com/fda-extends-evinacumab-indication-to-include-patients-with-hofh-ages-1-to-5-years/ The U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) for the treatment of children ages 1 to 5 years old with homozygous familial hypercholesterolemia (HoFH), supplemental to diet, exercise, and other lipid-lowering therapies. FDA Approves Elamipretide for Patients With Barth Syndrome https://checkrare.com/fda-approves-elamipretide-for-patients-with-barth-syndrome/ The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide HCI) for the treatment of patients with Barth syndrome. FDA Approves Updated REMS for IgA Nephropathy Treatment https://checkrare.com/fda-approves-updated-rems-for-iga-nephropathy-treatment/ The U.S. Food and Drug Administration (FDA) has approved the updated Risk Evaluation and Mitigation Strategy (REMS) for Filspari (sparsentan) for the treatment of IgA nephropathy (IgAN).