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

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). 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). Revumenib for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia https://checkrare.com/fda-approves-revumenib-for-the-treatment-of-relapsed-refractory-acute-myeloid-leukemia/ The U.S. FDA has approved Revuforj (revumenib) for the treatment of relapsed/refractory acute myeloid leukemia (AML). 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).