Wayrilz-Rilzabrutinib
Wayrilz is an oral reversible BTK inhibitor indicated for adults with chronic immune thrombocytopenia (ITP). It works by modulating immune pathways to increase platelet counts and reduce the risk of bleeding, offering a convenient twice-daily oral treatment option.
Forzinity-Elamipretide
Forzinity is a mitochondrial-targeted therapy for Barth syndrome in patients weighing 30 kg or more. It stabilizes mitochondrial function to improve energy production and muscle strength, administered via subcutaneous injection.
Inluriyo-Imlunestrant
Inluriyo is an oral selective estrogen receptor degrader (SERD) indicated for ER-positive, HER2-negative, ESR1-mutated advanced breast cancer. It degrades estrogen receptors to block tumor growth, offering a convenient daily oral option.
Palsonify-Paltusotine
Palsonify is an oral somatostatin receptor agonist approved for adults with acromegaly. It suppresses excess growth hormone and IGF-1 levels, providing a once-daily, non-injectable alternative to traditional therapies.
Rhapsido-Remibrutinib
Rhapsido is an oral BTK inhibitor indicated for chronic spontaneous urticaria (CSU). By blocking mast cell activation and histamine release, it effectively reduces hives and itch in patients unresponsive to antihistamines.
Qalsody-Tofersen
Qalsody is the first FDA‑approved antisense oligonucleotide treatment for amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. It reduces SOD1 protein synthesis by targeting SOD1 mRNA, addressing disease progression at a genetic level.
Miebo-Perfluorhexyloctane
Miebo is an eye drop that treats dry eye disease by reducing tear evaporation. It forms a protective layer on the eye to keep it moist and comfortable.
Elfabrio-Pegunigalsidase Alfa-Iwxj
Elfabrio is the first PEGylated enzyme replacement therapy approved by the FDA for adults with Fabry disease. It provides exogenous alpha‑galactosidase A to reduce globotriaosylceramide accumulation and improve organ function.
Veozah-Fezolinetant
Veozah is the first FDA‑approved non‑hormonal oral neurokinin 3 (NK3) receptor antagonist for moderate to severe vasomotor symptoms (hot flashes and night sweats) due to menopause. It blocks neurokinin B activity in the hypothalamus to regulate body temperature without estrogen .
Epkinly-Epcoritamab-Bysp
Epkinly is an injection used for certain types of blood cancer like large B-cell lymphoma. It helps immune cells find and destroy cancer cells.
