Organization Overview
Historical Acquisition Tree
Alternative names
cerliponase alfa (brineura) (2017 NDA)
elosulfase alfa (vimizim) (2014 NDA)
galsulfase (Naglazyme) (2005 NDA)
Laronidase (Aldurazyme) (2003 NDA)
pegvaliase (Palynziq) (2018 NDA)
sapropterin (kuvan) (2007 NDA)
valoctocogene roxaparvovec (Roctavian) (2023 NDA)
vosoritide (Voxzogo) (2021 NDA)
Achondroplasia (Phase 3)
Adenocarcinoma (Phase 1)
Albuminuria (Phase 2)
Anemia, Sickle Cell (Phase 2)
Autistic Disorder (Phase 3)
Blood Coagulation Disorders (Phase 2)
Carcinoma (Phase 1)
Carcinoma, Non-Small-Cell Lung (Phase 1)
Carcinoma, Ovarian Epithelial (Phase 1)
Coronary Artery Disease (Phase 2)
Coronary Disease (Phase 2)
Cystadenocarcinoma, Serous (Phase 1)
Disease (Phase 2)
Fallopian Tube Neoplasms (Phase 1)
Familial Primary Pulmonary Hypertension (Phase 1)
Glycogen Storage Disease Type II (Phase 3)
Hematologic Diseases (Phase 2)
Hemophilia A (Phase 3)
Hemostatic Disorders (Phase 2)
Hypertension (Phase 2)
Intermittent Claudication (Phase 2)
Kidney Diseases (Phase 2)
Lesch-Nyhan Syndrome (Phase 3)
Lung Neoplasms (Phase 1)
Mucopolysaccharidoses (Phase 4)
Mucopolysaccharidosis III (Phase 2)
Mucopolysaccharidosis IV (Phase 3)
Mucopolysaccharidosis VI (Phase 4)
Muscular Dystrophies (Phase 3)
Muscular Dystrophy, Duchenne (Phase 3)
Myocardial Ischemia (Phase 2)
Neoplasms (Phase 2)
Neuronal Ceroid-Lipofuscinoses (Phase 2)
Osteochondrodysplasias (Phase 3)
Ovarian Neoplasms (Phase 1)
Peripheral Arterial Disease (Phase 2)
Peripheral Vascular Diseases (Phase 2)
Peritoneal Neoplasms (Phase 1)
Phenylketonurias (Phase 4)
Recurrence (Phase 2)
Renal Insufficiency, Chronic (Phase 2)
Syndrome (Phase 4)