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 Treatment for Pheochromocytoma or Paraganglioma https://checkrare.com/fda-approves-treatment-for-pheochromocytoma-or-paraganglioma/ The U.S. Food and Drug Administration (FDA) approved Welireg (belzutifan) for patients ages 12 years and older with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma. FDA Approves Myasthenia Gravis Treatment Nipocalimab https://checkrare.com/fda-approves-myasthenia-gravis-treatment-nipocalimab/ The U.S. FDA has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG). The approval is for patients, 12 years of age and older, with anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) positive antibodies. FDA Approves Gene Therapy for Epidermolysis Bullosa https://checkrare.com/fda-approves-gene-therapy-for-epidermolysis-bullosa-2/ The U.S. FDA has approved Zevaskyn (prademagene zamikeracel or pz-cel) for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (EB). FDA Approves Hepatocellular Carcinoma Combination Therapy https://checkrare.com/fda-approves-hepatocellular-carcinoma-combination-therapy/ The U.S. FDA has approved nivolumab plus ipilimumab for first-line therapy in adult patients with unresectable or metastatic hepatocellular carcinoma (HCC).