FDA orphan drug designations for lysosomal storage disorders: a cross-sectional analysis
Purpose - To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs). - - Methods - Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with de...
Gespeichert in:
| Hauptverfasser: | , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
April 8, 2020
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| In: |
PLOS ONE
Year: 2020, Jahrgang: 15, Heft: 4 |
| ISSN: | 1932-6203 |
| DOI: | 10.1371/journal.pone.0230898 |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1371/journal.pone.0230898 Verlag, lizenzpflichtig, Volltext: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7141691/ 
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| Verfasserangaben: | Sven F. Garbade, Matthias Zielonka, Konstantin Mechler, Stefan Kölker, Georg F. Hoffmann, Christian Staufner, Eugen Mengel, Markus Ries |
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| 245 | 1 | 0 | |a FDA orphan drug designations for lysosomal storage disorders |b a cross-sectional analysis |c Sven F. Garbade, Matthias Zielonka, Konstantin Mechler, Stefan Kölker, Georg F. Hoffmann, Christian Staufner, Eugen Mengel, Markus Ries |
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| 520 | |a Purpose - To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs). - - Methods - Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descriptive and comparative statistics. - - Results - Between 1983 and 2019, 124 orphan drug designations were granted by the FDA for compounds intended to treat 28 lysosomal storage diseases. Orphan drug designations focused on Gaucher disease (N = 16), Pompe disease (N = 16), Fabry disease (N = 10), MPS II (N = 10), MPS I (N = 9), and MPS IIIA (N = 9), and included enzyme replacement therapies, gene therapies, and small molecules, and others. Twenty-three orphan drugs were approved for the treatment of 11 LSDs. Gaucher disease (N = 6), cystinosis (N = 5), Pompe disease (N = 3), and Fabry disease (N = 2) had multiple approvals, CLN2, LAL-D, MPS I, II, IVA, VI, and VII one approval each. This is an increase of nine more approved drugs and four more treatable LSDs (CLN2, MPS VII, LAL-D, and MPS IVA) since 2013. Mean time between orphan drug designation and FDA approval was 89.7 SD 55.00 (range 8-203, N = 23) months. - - Conclusions - The drug development pipeline for LSDs is growing and evolving, with increased focus on diverse small-molecule targets and gene therapy. CLN2 was the first and only LSD with an approved therapy directly targeted to the brain. Newly approved products included “me-too”-enzymes and innovative compounds such as the first pharmacological chaperone for the treatment of Fabry disease. | ||
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