Cipaglucosidase Alfa : First Approval
© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG..
Cipaglucosidase alfa (Pombiliti™) is a recombinant human acid α-glucosidase (GAA) product being developed by Amicus Therapeutics along with the enzyme stabilizer miglustat as a two-component therapy for Pompe disease. Pompe disease is a rare, inherited lysosomal disease caused by a deficiency of the enzyme GAA, which leads to accumulation of glycogen in various tissues. On 27 March 2023, cipaglucosidase alfa was approved in the EU as a long-term enzyme replacement therapy (ERT) used in combination with miglustat for the treatment of adults with late-onset Pompe disease. This article summarizes the milestones in the development of cipaglucosidase alfa leading to this first approval.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2023 |
|---|---|
| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:83 |
|---|---|
| Enthalten in: |
Drugs - 83(2023), 8 vom: 15. Juni, Seite 739-745 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Blair, Hannah A [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
1-Deoxynojirimycin |
|---|
| Anmerkungen: |
Date Completed 22.05.2023 Date Revised 22.05.2023 published: Print Citation Status MEDLINE |
|---|
| doi: |
10.1007/s40265-023-01886-5 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM356874540 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM356874540 | ||
| 003 | DE-627 | ||
| 005 | 20231226071404.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2023 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1007/s40265-023-01886-5 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1189.xml |
| 035 | |a (DE-627)NLM356874540 | ||
| 035 | |a (NLM)37184753 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Blair, Hannah A |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Cipaglucosidase Alfa |b First Approval |
| 264 | 1 | |c 2023 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 22.05.2023 | ||
| 500 | |a Date Revised 22.05.2023 | ||
| 500 | |a published: Print | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG. | ||
| 520 | |a Cipaglucosidase alfa (Pombiliti™) is a recombinant human acid α-glucosidase (GAA) product being developed by Amicus Therapeutics along with the enzyme stabilizer miglustat as a two-component therapy for Pompe disease. Pompe disease is a rare, inherited lysosomal disease caused by a deficiency of the enzyme GAA, which leads to accumulation of glycogen in various tissues. On 27 March 2023, cipaglucosidase alfa was approved in the EU as a long-term enzyme replacement therapy (ERT) used in combination with miglustat for the treatment of adults with late-onset Pompe disease. This article summarizes the milestones in the development of cipaglucosidase alfa leading to this first approval | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Review | |
| 650 | 7 | |a miglustat |2 NLM | |
| 650 | 7 | |a ADN3S497AZ |2 NLM | |
| 650 | 7 | |a 1-Deoxynojirimycin |2 NLM | |
| 650 | 7 | |a 19130-96-2 |2 NLM | |
| 650 | 7 | |a Glycogen |2 NLM | |
| 650 | 7 | |a 9005-79-2 |2 NLM | |
| 773 | 0 | 8 | |i Enthalten in |t Drugs |d 1976 |g 83(2023), 8 vom: 15. Juni, Seite 739-745 |w (DE-627)NLM00001611X |x 1179-1950 |7 nnns |
| 773 | 1 | 8 | |g volume:83 |g year:2023 |g number:8 |g day:15 |g month:06 |g pages:739-745 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1007/s40265-023-01886-5 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 83 |j 2023 |e 8 |b 15 |c 06 |h 739-745 | ||