The Food and Drug Administration (FDA) has approved Trikafta (elexacaftor/tezacaftor/ivacaftor; Vertex) for the treatment of patients aged 12 years and older with cystic fibrosis (CF) who have ≥1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Trikafta is a fixed-dose combination containing elexacaftor 100mg, tezacaftor 50mg, and ivacaftor 75mg that is co-packaged…
The Food and Drug Administration (FDA) has approved Nourianz (istradefylline; Kyowa Kirin) tablets as adjunctive treatment to levodopa/carbidopa in adult patients with Parkinson disease (PD) experiencing “off” episodes.
Results demonstrated inclisiran to have met both primary end points with a favorable safety profile.
Golodirsen is a phosphorodiamidate morpholino oligomer.
The FDA has granted Breakthrough Therapy designation to peginterferon lambda (Lambda; Eiger BioPharmaceuticals) for the treatment of hepatitis delta virus (HDV) infection.