Drugs / REVEGLUCOSIDASE ALFA
REVEGLUCOSIDASE ALFA
also known as
BMN 701 · Gilt-rhgaa · GILT-tagged Recombinant Human GAA · Reveglucosidasa alfa · ZC-701
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01230801 | Jan 2011 → Mar 2013 | glycogen storage disease due to acid maltase deficiency, late-onset | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT01435772 | Aug 2011 → Sep 2016 | glycogen storage disease due to acid maltase deficiency, late-onset | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT01924845 | Apr 2014 → Sep 2016 | glycogen storage disease due to acid maltase deficiency, late-onset | BioMarin Pharmaceutical | Terminated | No outcome recorded Stop: Business |
News releases announcing trial results or a regulatory action · 3
| Date | Issuer | Release |
|---|---|---|
| 2015-09-30 | BioMarin Pharmaceutical | Results BioMarin to Present Research Results on Duchenne Muscular Dystrophy and Pompe Disease Programs at the 20th International Congress of the World Muscle Society biomarin.com ↗
During an oral session, preliminary clinical efficacy and safety data from an existing extension study of reveglucosidase alfa (BMN 701) in patients with late onset Pompe disease will be presented. |
| 2014-02-13 | BioMarin Pharmaceutical | Results BioMarin Presents 15 Abstracts From Basic Research to Clinical Trials at Lysosomal Disease Network’s 10th Annual WORLDSymposium(TM) biomarin.com ↗
Preliminary Clinical Efficacy and Safety of BMN 701, GILT-tagged Recombinant Human Acid Alpha Glucosidase (rhGAA), in Late Onset Pompe Disease: Results of an Extension Study |
| 2013-09-17 | BioMarin Pharmaceutical | Regulatory BioMarin Provides Highlights Ahead of Research and Development Day biomarin.com ↗
BMN 701: Maximal Inspiratory Pressure (MIP) accepted by FDA as primary endpoint for Phase 3 Study in Late Onset Pompe Disease. |
All press releases naming this drug 5 releases
| Date | Issuer | Release |
|---|---|---|
| 2015-09-30 | BioMarin Pharmaceutical | BioMarin to Present Research Results on Duchenne Muscular Dystrophy and Pompe Disease Programs at the 20th International Congress of the World Muscle Society biomarin.com ↗ |
| 2014-05-27 | BioMarin Pharmaceutical | BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease biomarin.com ↗ |
| 2014-02-13 | BioMarin Pharmaceutical | BioMarin Presents 15 Abstracts From Basic Research to Clinical Trials at Lysosomal Disease Network’s 10th Annual WORLDSymposium(TM) biomarin.com ↗ |
| 2013-09-17 | BioMarin Pharmaceutical | BioMarin Provides Highlights Ahead of Research and Development Day biomarin.com ↗ |
| 2011-01-19 | BioMarin Pharmaceutical | BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease biomarin.com ↗ |
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | REVEGLUCOSIDASE ALFA | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707295 ↗ |
| Known as | BMN 701 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01924845 ↗ |
| Known as | Gilt-rhgaa | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707295 ↗ |
| Known as | GILT-tagged Recombinant Human GAA | “A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With...” NCT01924845 ↗ |
| Known as | Reveglucosidasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707295 ↗ |
| Known as | ZC-701 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707295 ↗ |
| Modality | Protein / enzyme biologic | “GILT-tagged recombinant human GAA” NCT01230801 ↗ |
| Route | Intravenous | “BMN 701 administered by intravenous infusion” NCT01230801 ↗ |