2025 Orphan Drugs: PDUFA Dates and FDA Approvals ++++ ++++

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 Nitisinone for the Treatment of Alkaptonuria https://checkrare.com/fda-approves-nitisinone-for-the-treatment-of-alkaptonuria/ The U.S. FDA has approved Harliku (nitisinone) for the treatment of adult patients with alkaptonuria. FDA Approves Dupixent for the Treatment of Bullous Pemphigoid https://checkrare.com/fda-approves-dupixent-for-the-treatment-of-bullous-pemphigoid/ The U.S. FDA has approved Dupixent (dupilumab) for the treatment of adult patients with bullous pemphigoid. FDA Approves Garadacimab for Hereditary Angioedema https://checkrare.com/fda-approves-garadacimab-for-hereditary-angioedema/ The U.S. FDA has approved Andembry (garadacimab) for the treatment of hereditary angioedema (HAE) in patients ages 12 years and older. CLINICAL PERSPECTIVES Patient Experience With Stevens-Johnson Syndrome https://checkrare.com/patient-experience-with-stevens-johnson-syndrome/ Katie Niemeyer, patient advocate, discusses her experience with Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN). Evolving Policy Landscapes for Rare Disease Access https://checkrare.com/evolving-policy-landscapes-for-rare-disease-access/ Deb Jennings, Head of North America Patient Services Operations at Kyowa Kirin, discusses evolving policy landscapes for rare disease access. The Role of Mental Health in Rare Disease Patient Outcomes https://checkrare.com/the-role-of-mental-health-in-rare-disease-patient-outcomes/ Sumira Riaz, PhD, Health Psychologist & Patient Engagement Consultant at Unboxed Psychology, discusses the role of mental health in rare disease patient outcomes. The Undiagnosed Disease Network https://checkrare.com/the-undiagnosed-disease-network/ Kimberly LeBlanc, Genetic Counselor, Director of the Undiagnosed Diseases Network (UDN) Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases.