The Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for sepiapterin as a treatment for pediatric and adult patients living with phenylketonuria (PKU).
Trastuzumab deruxtecan is a HER2-directed antibody and topoisomerase inhibitor conjugate currently marketed under the brand name Enhertu (fam-trastuzumab deruxtecan-nxki) for the treatment of breast ...
The Food and Drug Administration (FDA) has granted Fast Track designation to zilganersen for the treatment of Alexander disease, a rare type of leukodystrophy.