Gomekli-Mirdametinib
Gomekli treats adults and children (≥2 years) with NF1-related plexiform neurofibromas that can’t be fully removed. It inhibits MEK enzymes to slow tumor growth and reduce tumor size.
Romvimza-Vimseltinib
Romvimza is a tablet for adults with tenosynovial giant cell tumor when surgery might worsen joint function. It blocks CSF-1R to shrink tumors and ease symptoms.
Blujepa-Gepotidacin
Blujepa treats uncomplicated urinary tract infections in females and adolescents with more than 12 years and more than or equal to 40 kg. It’s an oral antibiotic in a new class that combats common UTI bacteria and tackles antibiotic resistance.
Qfitlia-Fitusiran
Qfitlia is a subcutaneous therapy approved for routine prevention of bleeding in patients aged 12 and older with hemophilia A or B, with or without inhibitors. It lowers antithrombin to rebalance clotting and has been shown to reduce annual bleeding rates by around 90%
Vanrafia-Atrasentan
Vanrafia is an oral treatment for adults with primary IgA nephropathy at risk of rapid disease progression. It reduces proteinuria and helps protect kidney function by blocking endothelin A receptors.
Bizengri-Zenocutuzumab-Zbco
Bizengri is an IV antibody therapy for adults with advanced non-small cell lung cancer or pancreatic cancer driven by NRG1 gene fusions, used after standard treatments fail. It blocks the fusion protein’s signaling to help shrink tumors and control disease progression
Alyftrek-Vanzacaftor, Tezacaftor, And DeutIVacaftor
Alyftrek is a daily oral treatment for cystic fibrosis in patients aged 6 and older with the F508del mutation or other responsive CFTR variants. It helps restore CFTR protein function to improve lung performance and reduce disease flare-ups.
Ensacove-Ensartinib
Ensacove is an oral kinase inhibitor for adults with ALK-positive, locally advanced or metastatic non-small cell lung cancer who haven’t previously received ALK-targeted therapy. It blocks ALK-driven signaling to help control tumor growth and delay disease progression.
Tryngolza-Olezarsen
Tryngolza is a subcutaneous injection for adults with familial chylomicronemia syndrome. It reduces APOC-III protein levels to lower dangerously high triglycerides and prevent pancreatitis.
Vyloy-Zolbetuximab-Clzb
Vyloy is used with fluoropyrimidine and platinum-based chemotherapy as the first-line treatment for HER2-negative gastric or gastroesophageal junction cancer, which cannot be removed by surgery or has spread and tests positive for claudin (CLDN)18.2.
