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. Below is the list of important regulatory dates for all orphan drugs for 2026. 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. FDA Approves Gene Therapy (Waskyra) 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 Selumetinib to Treat Patients With Neurofibromatosis Type 1 https://checkrare.com/fda-approves-selumetinib-to-treat-patients-with-neurofibromatosis-type-1/ The U.S. FDA has approved Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).