2025 Orphan Drugs: PDUFA Dates and FDA Approvals By: Peter Ciszewski|Published on: Updated on: Jan 1, 2026 ++++

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. 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. Hyrnua for HER2-Positive Non-Small Cell Lung Cancer Gets FDA Approval https://checkrare.com/fda-approves-hyrnua-for-her2-positive-non-small-cell-lung-cancer/ The U.S. FDA has approved Hyrnua (sevabertinib) for the treatment of adults with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) with HER2 tyrosine kinase domain activating mutations. FDA Expands Indication of Sotatercept for Patients With Pulmonary Arterial Hypertension https://checkrare.com/fda-expands-indication-of-sotatercept-for-patients-with-pulmonary-arterial-hypertension/ The U.S. FDA has approved an update to the product label of Winrevair (sotatercept) for the treatment of adults with pulmonary arterial hypertension (PAH).