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 (LSD’s). - Methods Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descript...
Saved in:
| Main Authors: | , , , , , , , |
|---|---|
| Format: | Article (Journal) Chapter/Article |
| Language: | English |
| Published: |
January 14, 2020
|
| In: |
medRxiv
Year: 2020, Pages: 1-29 |
| DOI: | 10.1101/2020.01.05.20016568 |
| Online Access: | Verlag, kostenfrei, Volltext: https://doi.org/10.1101/2020.01.05.20016568 Verlag, kostenfrei, Volltext: https://www.medrxiv.org/content/10.1101/2020.01.05.20016568v2 
|
| Author Notes: | Sven F. Garbade PhD, Matthias Zielonka MD, Konstantin Mechler MD, Stefan Kölker MD, Georg F. Hoffmann MD, Christian Staufner MD, Eugen Mengel MD and Markus Ries MD PhD |
MARC
| LEADER | 00000caa a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1800234554 | ||
| 003 | DE-627 | ||
| 005 | 20240711154217.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 220425s2020 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1101/2020.01.05.20016568 |2 doi | |
| 035 | |a (DE-627)1800234554 | ||
| 035 | |a (DE-599)KXP1800234554 | ||
| 035 | |a (OCoLC)1341458973 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 33 |2 sdnb | ||
| 100 | 1 | |a Garbade, Sven |d 1971- |e VerfasserIn |0 (DE-588)129234362 |0 (DE-627)707186889 |0 (DE-576)297554263 |4 aut | |
| 245 | 1 | 0 | |a FDA orphan drug designations for lysosomal storage disorders |b a cross sectional analysis |c Sven F. Garbade PhD, Matthias Zielonka MD, Konstantin Mechler MD, Stefan Kölker MD, Georg F. Hoffmann MD, Christian Staufner MD, Eugen Mengel MD and Markus Ries MD PhD |
| 264 | 1 | |c January 14, 2020 | |
| 300 | |a 29 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Gesehen am 25.04.2022 | ||
| 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 (LSD’s). - 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 LSD’s (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 development pipeline is growing and evolving into diversified small molecules 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. | ||
| 700 | 1 | |a Zielonka, Matthias |d 1983- |e VerfasserIn |0 (DE-588)1056891297 |0 (DE-627)79418670X |0 (DE-576)412871637 |4 aut | |
| 700 | 1 | |a Mechler, Konstantin |d 1987- |e VerfasserIn |0 (DE-588)1076373658 |0 (DE-627)834806304 |0 (DE-576)445254831 |4 aut | |
| 700 | 1 | |a Kölker, Stefan |e VerfasserIn |0 (DE-588)1022937758 |0 (DE-627)717335771 |0 (DE-576)366197568 |4 aut | |
| 700 | 1 | |a Hoffmann, Georg F. |d 1957- |e VerfasserIn |0 (DE-588)115652868 |0 (DE-627)077386116 |0 (DE-576)261230042 |4 aut | |
| 700 | 1 | |a Staufner, Christian |d 1980- |e VerfasserIn |0 (DE-588)141097515 |0 (DE-627)703880950 |0 (DE-576)321299469 |4 aut | |
| 700 | 1 | |a Mengel, Eugen |d 1963- |e VerfasserIn |0 (DE-588)135623901 |0 (DE-627)56860427X |0 (DE-576)300550545 |4 aut | |
| 700 | 1 | |a Ries, Markus |d 1971- |e VerfasserIn |0 (DE-588)136385338 |0 (DE-627)582068479 |0 (DE-576)300993358 |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t medRxiv |d Cold Spring Harbor : Cold Spring Harbor Laboratory, 2019 |g (2020), Artikel-ID 2020.01.05.20016568, Seite 1-29 |h Online-Ressource |w (DE-627)1738046478 |w (DE-600)3044535-8 |7 nnas |a FDA orphan drug designations for lysosomal storage disorders a cross sectional analysis |
| 773 | 1 | 8 | |g year:2020 |g elocationid:2020.01.05.20016568 |g pages:1-29 |g extent:29 |a FDA orphan drug designations for lysosomal storage disorders a cross sectional analysis |
| 856 | 4 | 0 | |u https://doi.org/10.1101/2020.01.05.20016568 |x Verlag |x Resolving-System |z kostenfrei |3 Volltext |
| 856 | 4 | 0 | |u https://www.medrxiv.org/content/10.1101/2020.01.05.20016568v2 |x Verlag |z kostenfrei |3 Volltext |
| 951 | |a AR | ||
| 992 | |a 20220425 | ||
| 993 | |a Article | ||
| 994 | |a 2020 | ||
| 998 | |g 136385338 |a Ries, Markus |m 136385338:Ries, Markus |d 910000 |d 910500 |d 50000 |e 910000PR136385338 |e 910500PR136385338 |e 50000PR136385338 |k 0/910000/ |k 1/910000/910500/ |k 0/50000/ |p 8 |y j | ||
| 998 | |g 141097515 |a Staufner, Christian |m 141097515:Staufner, Christian |d 910000 |d 910500 |e 910000PS141097515 |e 910500PS141097515 |k 0/910000/ |k 1/910000/910500/ |p 6 | ||
| 998 | |g 115652868 |a Hoffmann, Georg F. |m 115652868:Hoffmann, Georg F. |d 910000 |d 910500 |e 910000PH115652868 |e 910500PH115652868 |k 0/910000/ |k 1/910000/910500/ |p 5 | ||
| 998 | |g 1022937758 |a Kölker, Stefan |m 1022937758:Kölker, Stefan |d 910000 |d 910500 |d 50000 |e 910000PK1022937758 |e 910500PK1022937758 |e 50000PK1022937758 |k 0/910000/ |k 1/910000/910500/ |k 0/50000/ |p 4 | ||
| 998 | |g 1076373658 |a Mechler, Konstantin |m 1076373658:Mechler, Konstantin |d 60000 |e 60000PM1076373658 |k 0/60000/ |p 3 | ||
| 998 | |g 1056891297 |a Zielonka, Matthias |m 1056891297:Zielonka, Matthias |d 910000 |d 910500 |e 910000PZ1056891297 |e 910500PZ1056891297 |k 0/910000/ |k 1/910000/910500/ |p 2 | ||
| 998 | |g 129234362 |a Garbade, Sven |m 129234362:Garbade, Sven |d 910000 |d 910500 |e 910000PG129234362 |e 910500PG129234362 |k 0/910000/ |k 1/910000/910500/ |p 1 |x j | ||
| 999 | |a KXP-PPN1800234554 |e 4120787400 | ||
| BIB | |a Y | ||
| JSO | |a {"recId":"1800234554","language":["eng"],"physDesc":[{"extent":"29 S."}],"name":{"displayForm":["Sven F. Garbade PhD, Matthias Zielonka MD, Konstantin Mechler MD, Stefan Kölker MD, Georg F. Hoffmann MD, Christian Staufner MD, Eugen Mengel MD and Markus Ries MD PhD"]},"id":{"doi":["10.1101/2020.01.05.20016568"],"eki":["1800234554"]},"origin":[{"dateIssuedDisp":"January 14, 2020","dateIssuedKey":"2020"}],"person":[{"display":"Garbade, Sven","role":"aut","family":"Garbade","given":"Sven"},{"given":"Matthias","family":"Zielonka","display":"Zielonka, Matthias","role":"aut"},{"given":"Konstantin","family":"Mechler","display":"Mechler, Konstantin","role":"aut"},{"given":"Stefan","display":"Kölker, Stefan","role":"aut","family":"Kölker"},{"given":"Georg F.","family":"Hoffmann","display":"Hoffmann, Georg F.","role":"aut"},{"given":"Christian","role":"aut","display":"Staufner, Christian","family":"Staufner"},{"given":"Eugen","family":"Mengel","role":"aut","display":"Mengel, Eugen"},{"given":"Markus","display":"Ries, Markus","role":"aut","family":"Ries"}],"title":[{"subtitle":"a cross sectional analysis","title_sort":"FDA orphan drug designations for lysosomal storage disorders","title":"FDA orphan drug designations for lysosomal storage disorders"}],"relHost":[{"type":{"media":"Online-Ressource","bibl":"edited-book"},"origin":[{"publisher":"Cold Spring Harbor Laboratory","dateIssuedKey":"2019","dateIssuedDisp":"[2019?]-","publisherPlace":"Cold Spring Harbor"}],"title":[{"title":"medRxiv","title_sort":"medRxiv","subtitle":"the preprint server for health sciences"}],"note":["medRxiv (pronounced \"med-archive\") is a free online archive and distribution server for complete but unpublished manuscripts (preprints) in the medical, clinical, and related health sciences. Preprints are preliminary reports of work that have not been certified by peer review. They should not be relied on to guide clinical practice or health-related behavior and should not be reported in news media as established information","Gesehen am 06.11.2020"],"disp":"FDA orphan drug designations for lysosomal storage disorders a cross sectional analysismedRxiv","part":{"text":"(2020), Artikel-ID 2020.01.05.20016568, Seite 1-29","extent":"29","year":"2020","pages":"1-29"},"language":["eng"],"physDesc":[{"extent":"Online-Ressource"}],"recId":"1738046478","id":{"eki":["1738046478"],"zdb":["3044535-8"]}}],"type":{"bibl":"chapter","media":"Online-Ressource"},"note":["Gesehen am 25.04.2022"]} | ||
| SRT | |a GARBADESVEFDAORPHAND1420 | ||