Acoramidis, an oral therapy, is now approved to reduce cardiovascular-related deaths and hospitalizations in adults with hereditary or wild-type ATTR-CM.
The FDA has approved concizumab-mtci, a daily subcutaneous treatment to reduce bleeding for patients with hemophilia who have clotting factor inhibitors.
Crinecerfont, a first-in-class therapy, improved androgen control and reduced reliance on high-dose glucocorticoids for patients with congenital adrenal hyperplasia.
Two randomized, double-blind studies demonstrated that a new antipsychotic treatment for adult patients with schizophrenia may lead to a meaningful reduction in symptoms.