Crenessity-Crinecerfont
Crenessity is used to treat classic congenital adrenal hyperplasia. It is administered together with glucocorticoids to control androgen levels in adults and children 4 years of age and older with classic congenital adrenal hyperplasia.
Iomervu-Iomeprol
Iomervu is an injectable contrast agent for CT scans and angiography in adults and children. It enhances artery and organ imaging by providing clear, safe visualization of vascular structures.
Unloxcyt-Cosibelimab-Ipdl
Unloxcyt is an IV anti–PD-L1 antibody for adults with advanced or metastatic cutaneous squamous cell carcinoma who are not surgical or radiation candidates. It helps the immune system fight cancer by blocking PD-L1, with a documented response in nearly half of patients.
Hernexeos-Zongertinib
Hernexeos is an oral tyrosine kinase inhibitor used to treat adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors harbor HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations. Treatment is indicated following prior systemic therapy, and eligibility requires confirmation of the HER2 mutation via an FDA-approved test.
Brinsupri-Brensocatib
Brinsupri is an oral, once-daily dipeptidyl peptidase-1 (DPP1) inhibitor for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adults and adolescents aged 12 years and older. It represents the first FDA-approved targeted therapy for this chronic, progressive lung disease, directly addressing neutrophil-driven inflammation rather than just symptom management.
Modeyso-Dordaviprone
Modeyso is an oral protease activator approved for adults and children with recurrent H3 K27M-mutant diffuse midline glioma (DMG), a rare and aggressive brain tumor. By activating mitochondrial caseinolytic protease P (ClpP) and antagonizing dopamine D2 receptors, it induces stress-response pathways, apoptosis, and restoration of histone H3 trimethylation, ultimately slowing tumor growth. Taken once weekly […]
Vizz-Aceclidine
Vizz is the first and only aceclidine-based eye drop approved for adults with presbyopia. Presbyopia is an age-related blurry near vision. By selectively contracting the iris sphincter muscle to create a pinhole effect and achieve a sub-2 mm pupil—without significant ciliary muscle stimulation—it extends depth of focus to provide fast, steroid-free relief of near-vision blur […]
Anzupgo-Delgocitinib
Anzupgo cream is the first and only topical pan-JAK inhibitor approved for adults with moderate to severe chronic hand eczema. By targeting the JAK-STAT pathway, it provides effective, steroid-free relief from inflammation, itching, and skin damage, giving patients a safer and more convenient alternative to traditional treatments.
Sephience-Sepiapterin
Sephience is the first and only oral phenylalanine hydroxylase activator approved for the treatment of hyperphenylalaninemia in adults and children with sepiapterin-responsive phenylketonuria. By delivering sepiapterin, which converts into tetrahydrobiopterin and also prevents PAH misfolding, it enhances enzyme function to reduce blood phenylalanine levels, offering patients a steroid-free, non-invasive, and more convenient alternative to dietary […]
Ekterly-Sebetralstat
Ekterly is the first and only oral, on-demand therapy for hereditary angioedema (HAE) attacks in patients aged 12 and older. It works by inhibiting plasma kallikrein, preventing excessive bradykinin production to reduce swelling rapidly. Approved in the U.S. and U.K. in 2025, Ekterly offers fast relief, convenient oral dosing, and a major advance over injectable […]
