2026 Orphan Drugs: PDUFA Dates and FDA Approvals +++++

FDA NEWS 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-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. Aqvesme (Mitapivat) Approved for Treatment of Anemia in Alpha- and Beta-Thalassemia https://checkrare.com/fda-approves-aqvesme-mitapivat-for-treating-anemia-in-alpha-and-beta-thalassemia/ The U.S. FDA has approved Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia. FDA Approves Obstructive Hypertrophic Cardiomyopathy Treatment https://checkrare.com/fda-approves-obstructive-hypertrophic-cardiomyopathy-treatment/ The U.S. FDA has approved Myqorzo (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Gene Therapy (Waskyra) Approved for Patients with Wiskott-Aldrich Syndrome https://checkrare.com/fda-approves-gene-therapy-waskyra-for-patients-with-wiskott-aldrich-syndrome/ The U.S. FDA has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS). FDA Approves Oral Prophylactic Therapy (berotralstat) for Pediatric Patients With Hereditary Angioedema https://checkrare.com/fda-approves-oral-prophylactic-therapy-berotralstat-for-pediatric-patients-with-hereditary-angioedema/ The U.S. FDA has approved Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to 12 years of age.