TY  - JOUR
AU  - Johann, Pascal
AU  - Lenz, Dominic
AU  - Ries, Markus
TI  - The drug development pipeline for glioblastoma-A cross sectional assessment of the FDA Orphan Drug Product designation database.
JO  - PLOS ONE
VL  - 16
IS  - 7
SN  - 1932-6203
CY  - San Francisco, California, US
PB  - PLOS
M1  - DKFZ-2021-01552
SP  - e0252924 -
PY  - 2021
N1  - #EA:B062#
AB  - Glioblastoma (GBM) is the most common malignant brain tumour among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the molecular makeup of this disease has been increasing rapidly. However, the clinical outcomes in GBM patients with currently available therapies are still dismal. An insight into the current drug development pipeline for GBM is therefore of particular interest.To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat GBM.Quantitative cross-sectional analysis of the U.S. Food and Drug Administration Orphan Drug Product database between 1983 and 2020. STROBE criteria were respected.Four orphan drugs out of 161 (2,4
LB  - PUB:(DE-HGF)16
C6  - pmid:34234357
DO  - DOI:10.1371/journal.pone.0252924
UR  - https://inrepo02.dkfz.de/record/169798
ER  -