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. FDA Approves Blenrep Combination Therapy for Multiple Myeloma https://checkrare.com/fda-approves-blenrep-combination-therapy-for-multiple-myeloma/ The U.S. FDA has approved Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BVd) for the treatment of adult patients with relapsed/refractory multiple myeloma (MM). FDA Approves Epioxa to Treat Keratoconus https://checkrare.com/fda-approves-epioxa-to-treat-keratoconus/ The U.S. Food and Drug Administration (FDA) has approved Epioxa (riboflavin 5’-phosphate ophthalmic solution) for the treatment of patients with keratoconus. FDA Approves Obinutuzumab for Patients With Lupus Nephritis https://checkrare.com/fda-approves-obinutuzumab-for-patients-with-lupus-nephritis/ The U.S. FDA has approved Gazyva/Gazyvaro (obinutuzumab) for the treatment of adults with active lupus nephritis who are receiving standard of care. FDA Approves Nerandomilast To Treat Patients With Idiopathic Pulmonary Fibrosis https://checkrare.com/fda-approves-nerandomilast-to-treat-patients-with-idiopathic-pulmonary-fibrosis/ The U.S. FDA has approved Jascayd (nerandomilast) tablets for the treatment of patients with idiopathic pulmonary fibrosis (IPF). FDA Extends Evinacumab Indication to Include Patients With HoFH Ages 1 to 5 Years https://checkrare.com/fda-extends-evinacumab-indication-to-include-patients-with-hofh-ages-1-to-5-years/ The U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) for the treatment of children ages 1 to 5 years old with homozygous familial hypercholesterolemia (HoFH), supplemental to diet, exercise, and other lipid-lowering therapies. FDA Approves Paltusotine for Treatment of Adults With Acromegaly https://checkrare.com/fda-approves-paltusotine-for-treatment-of-adults-with-acromegaly/ The U.S. Food and Drug Administration (FDA) has approved Palsonify (paltusotine) for the first-line treatment of adults with acromegaly with inadequate response to surgery and/or for whom surgery is not an option.