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.
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.
Orlynvah-Sulopenem Etzadroxil And Probenecid
Orlynvah is an oral tablet for adult women with uncomplicated urinary tract infections caused by E. coli, K. pneumoniae, or P. mirabilis, especially for those with limited or no alternative oral antibiotic options. It combines sulopenem (an antibiotic) with probenecid, which helps increase sulopenem levels, delivering strong antibacterial activity comparable or superior to ciprofloxacin and […]
Revuforj-Revumenib Citrate
Revuforj is a drug used to treat relapsed or refractory acute leukemia. The treatment of acute leukemia is done with a lysine methyltransferase 2A gene translocation (KMT2A) in adults. This drug is also indicated for children 1 year and older whose disease has come back or has not improved after previous treatments.
