Rare Disease News, Insight, and Clinical Perspective ++++++

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. Below is the list of important regulatory dates for all orphan drugs for 2025. The FDA Approves Sepiapterin for Treatment of Phenylketonuria https://checkrare.com/the-fda-approves-sepiapterin-for-treatment-of-phenylketonuria/ The U.S. Food and Drug Administration (FDA) has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU). The FDA Approves Pegcetacoplan for Treatment of Rare Kidney Disorders (C3G and IC-MPGN) https://checkrare.com/the-fda-approves-pegcetacoplan-for-treatment-of-rare-kidney-disorders-c3g-and-ic-mpgn/ The U.S. Food and Drug Administration (FDA) has approved Empaveli (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients ages 12 years and older to reduce proteinuria. FDA Approves Lonapegsomatropin for Treatment of Adults With Growth Hormone Deficiency https://checkrare.com/fda-approves-lonapegsomatropin-for-treatment-of-adults-with-growth-hormone-deficiency/ The U.S. Food and Drug Administration (FDA) has approved Skytrofa (lonapegsomatropin-tcgd; TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). The FDA Approves Avatrombopag to Treat Young Children with ITP https://checkrare.com/the-fda-approves-avatrombopag-to-treat-young-children-with-itp/ The U.S. Food and Drug Administration (FDA) has approved Doptelet (avatrombopag) for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients one year and older who have had insufficient response to prior therapy. The FDA Approves Avatrombopag to Treat Young Children with ITP https://checkrare.com/the-fda-approves-avatrombopag-to-treat-young-children-with-itp/ The U.S. Food and Drug Administration (FDA) has approved Doptelet (avatrombopag) for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients one year and older who have had insufficient response to prior therapy.