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 Hyrnua for HER2-Positive Non-Small Cell Lung Cancer 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. 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). Updates to Prescribing Information for Elevidys in Treatment of Duchenne Muscular Dystrophy https://checkrare.com/updates-to-prescribing-information-for-elevidys-in-treatment-of-duchenne-muscular-dystrophy/ The U.S. FDA has updated the prescribing information for Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). Approval of Treatment for Patients With Familial Chylomicronemia Syndrome (FCS) https://checkrare.com/approval-of-treatment-for-patients-with-familial-chylomicronemia-syndrome-fcs/ The U.S. FDA has approved Redemplo (plozasiran) for the treatment of adult patients with familial chylomicronemia syndrome (FCS). Ziftomenib for Relapsed/Refractory NPM1-Mutated Acute Myeloid Leukemia https://checkrare.com/fda-approves-ziftomenib-for-relapsed-refractory-npm1-mutated-acute-myeloid-leukemia/ The U.S. FDA has approved Komzifti (ziftomenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with susceptible NPM1 mutation. Darzalex Faspro for Patients With High-Risk Smoldering Multiple Myeloma https://checkrare.com/fda-approves-darzalex-faspro-for-patients-with-high-risk-smoldering-multiple-myeloma/ The U.S. FDA has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM). FDA Approves First Treatment for Thymidine Kinase 2 Deficiency https://checkrare.com/fda-approves-first-treatment-for-thymidine-kinase-2-deficiency/ The U.S. FDA has approved Kygevvi for the treatment of adults and pediatric patients, with an age of symptom onset on or before 12 years, with thymidine kinase 2 deficiency (TK2d).