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 […]
Lynozyfic-Linvoseltamab-Gcpt
Lynozyfic is a monotherapy for adults with relapsed and refractory multiple myeloma after ≥3 prior therapies. This bispecific T-cell engager bridges CD3 on T-cells with BCMA protein on myeloma cells to activate cancer-cell killing. FDA and EMA approved for heavily pretreated patients who have exhausted standard treatment options.
Zegfrovy-Sunvozertinib
Zegfrovy is a significant breakthrough in precision oncology for patients with non-small cell lung cancer (NSCLC) who have EGFR exon 20 insertion mutations. It sets a new benchmark for patients with few therapy alternatives by combining clinically significant efficacy, oral convenience, and highly selective EGFR inhibition. Its quick approval highlights the critical need for efficient […]
Ibtrozi-Taletrectinib
Ibtrozi is a targeted oral therapy for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer. This selective ROS1 tyrosine kinase inhibitor received FDA approval in June 2025 with breakthrough therapy designation. Features enhanced CNS penetration for improved efficacy against brain metastases compared to earlier ROS1 inhibitors.
Andembry-Garadacimab-Gxii
Andembry is a preventive therapy for hereditary angioedema (HAE) in adults and adolescents 12+ years. This selective Factor XIIa inhibitor targets the top of the inflammatory cascade with once-monthly dosing. FDA-approved in June 2025, offering the first monthly prophylactic HAE treatment targeting Factor XIIa.
Tryptyr-Acoltremon
Tryptyr is an oral neuroactive steroid modulator designed to treat dry eye disease. It acts on nervous system pathways to relieve symptoms and improve eye comfort.
