drugset
Drugs / alglucosidase alfa
last change Nov 2024 re-read 9 days ago

alglucosidase alfa

also known as MYOZYME · rhGAA · GZ419829 · .alpha.-glucosidase · Acid .alpha.-glucosidase · alg · Alglucosidasa alfa · Alglucosidase · +9 more

Protein / enzyme biologic targets GAA

Developed for
glycogen storage disease II · glycogen storage disease due to acid maltase deficiency, late-onset · glycogen storage disease due to acid maltase deficiency, infantile onset
Investigated by
Genzyme, a Sanofi Company · Erasmus Medical Center

Regulatory milestones approvals, filings & regulatory actions · 3 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) LUMIZYME — 2014-08-01 fda.gov ↗
Approved US (FDA) MYOZYME — 2006-04-28 fda.gov ↗
Approved EU (EMA) Myozyme — 2006-03-28 europa.eu ↗

Trials 22

today
EU approved 2006
US approved 2006
NCT06575829 · Ph 4 NCT04676373 · Ph 4 NCT03687333 · Ph 4 NCT03019406 · Ph 2 NCT01410890 · Ph 4 NCT01526785 · Ph 4 NCT01288027 · Ph 4 NCT00701129 · Ph 4 NCT00486889 · Ph 4 NCT00483379 · Ph 4 NCT00455195 · Ph 4 NCT00268944 · Ph 3 NCT00125879 · Ph 2/3 NCT00250939 · Ph 2 NCT00074932 · not/applicable NCT00074919 NCT00763932 · Ph 2 NCT00059280 · Ph 2/3 NCT00765414 · Ph 2 NCT00053573 · Ph 1/2 NCT00051935 · Ph 2
2003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/21 trial
Phase 1/2 NCT00053573 Feb 2003 → Jul 2006 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 25 trials
Phase 2 NCT03019406 Oct 2017 → Sep 2019 overdue glycogen storage disease due to acid maltase deficiency, infantile onset Genzyme, a Sanofi Company Active not recruiting No outcome recorded
Phase 2 NCT00250939 Feb 2005 → Jul 2006 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed No outcome recorded
Phase 2 NCT00763932 Apr 2003 → Jun 2006 glycogen storage disease due to acid maltase deficiency, infantile onset Genzyme, a Sanofi Company Completed No outcome recorded
Phase 2 NCT00765414 Apr 2003 → Apr 2006 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed No outcome recorded
Phase 2 NCT00051935 Jan → Apr 2003 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 2/32 trials
Phase 2/3 NCT00125879 Jun 2005 → Jun 2006 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 2/3 NCT00059280 Apr 2003 → Jun 2005 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 31 trial
Phase 3 NCT00268944 Dec 2005 → Mar 2007 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed No outcome recorded
Phase 410 trials · 1 missed
Phase 4 NCT06575829 Oct 2024 → Dec 2026 expected glycogen storage disease due to acid maltase deficiency, late-onset Erasmus Medical Center Not yet recruiting No outcome recorded
Phase 4 NCT04676373 Mar 2021 → Jul 2024 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT03687333 Dec 2018 → Dec 2020 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT01410890 Nov 2014 → Nov 2020 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT01526785 Mar 2012 → Dec 2014 glycogen storage disease II Genzyme, a Sanofi Company Terminated No outcome recorded
Phase 4 NCT01288027 Jun 2011 → Dec 2013 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed Missed primary
Phase 4 NCT00701129 Oct 2009 → Mar 2013 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT00486889 Aug 2008 → Nov 2021 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT00483379 May 2007 → Dec 2009 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT00455195 Mar 2007 → Oct 2008 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed No outcome recorded
Phase not applicable1 trial
— NCT00074932 Nov 2004 → Aug 2006 glycogen storage disease II Genzyme, a Sanofi Company Completed No outcome recorded
Phase not stated2 trials
— NCT00074919 Dec 2003 → Feb 2007 glycogen storage disease II Genzyme, a Sanofi Company Approved for marketing No outcome recorded
— NCT00520143 — glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Approved for marketing No outcome recorded
Comparator or background therapy7 trials
Phase 1/2 NCT02898753 Comparator Jun 2017 → Mar 2020 glycogen storage disease due to acid maltase deficiency, late-onset Valerion Therapeutics, LLC Terminated No outcome recorded Stop: Business
Phase 2 NCT01380743 Background Oct 2011 → Jan 2013 glycogen storage disease II Amicus Therapeutics Completed No outcome recorded
Phase 3 NCT03729362 Comparator Dec 2018 → Dec 2020 glycogen storage disease due to acid maltase deficiency, late-onset Amicus Therapeutics Completed Missed primary
Phase 3 NCT02782741 Comparator Nov 2016 → Mar 2020 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed Met primary
Phase 3 NCT00158600 Comparator Sep 2005 → Sep 2007 glycogen storage disease due to acid maltase deficiency, late-onset Genzyme, a Sanofi Company Completed No outcome recorded

News releases announcing trial results or a regulatory action · 1

DateIssuerRelease
2024-02-05 Sanofi Results Media Update: New data demonstrate clinical safety, benefit and durability of Nexviazyme® across a wide-range of Pompe disease patient groups sanofi.com ↗
In the comparative COMET study, fewer LOPD patients in the avalglucosidase alfa group reported at least 1 IAR (13/51 [25.5%]) in comparison to the alglucosidase alfa group (16/49 [32.7%]).

All press releases naming this drug 1 release

DateIssuerRelease

Evidence & citations 22 cited values

Every value below carries the sentence it was read from. 34 sources stand behind the page.

FieldValueCited text
Known as alglucosidase alfa “All eligible patients with late-onset Pompe disease will be treated with alglucosidase alfa 20 mg/kg once every 4 weeks for 9 months.” NCT06575829 ↗
19

“alglucosidase alfa” NCT01380743 ↗

NCT01410890 ↗

NCT03687333 ↗

NCT03729362 ↗

NCT00074919 ↗

NCT00520143 ↗

NCT01526785 ↗

NCT01288027 ↗

NCT00158600 ↗

NCT03019406 ↗

Show 9 more sources
Known as .alpha.-glucosidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as Acid .alpha.-glucosidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as alg “The first approved ERT for LOPD was alglucosidase alfa (alg).” PMID 39535661 ↗ Nov 2024
Known as Alglucosidasa alfa ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as Alglucosidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as Alglucosidase alfa (genetical recombination) ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as Alpha-glucopyranosidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as Glucosidase, alpha- ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as GZ419829 “Alglucosidase alfa (GZ419829)” NCT02782741 ↗
1

“Alglucosidase alfa (GZ419829)” NCT03019406 ↗
Known as Lumizyme ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as MYOZYME “ALGLUCOSIDASE ALFA (MYOZYME)” NCT03687333 ↗
8

NCT00053573 ↗

NCT00059280 ↗

NCT00268944 ↗

NCT00074932 ↗

NCT00250939 ↗

NCT00763932 ↗

NCT00125879 ↗

NCT00765414 ↗
Known as Ndi 709 ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Known as recombinant human acid alpha-glucosidase “This study is being conducted to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme...” NCT00051935 ↗
Known as recombinant human alpha-glucosidase “recombinant human alpha-glucosidase” NCT01380743 ↗
Known as recombinant human GAA “Alglucosidase alfa (recombinant human GAA (rhGAA)) received approval in 2006 as a treatment for Pompe disease at the 160 L production scale.” PMID 29565424 ↗ Mar 2018
Known as rhGAA “This study is being conducted to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme...” NCT00051935 ↗
4

“Alglucosidase alfa (recombinant human GAA (rhGAA)) received approval in 2006 as a treatment for Pompe disease at the 160 L production scale.” PMID 29565424 ↗ Mar 2018

“alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])” NCT00520143 ↗

“Drug: RhGAA” NCT02898753 ↗

NCT01380743 ↗
Known as Transglucosidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201824 ↗
Action Restore “enzyme replacement therapy” PMID 21543987 ↗ Jul 2011
Modality Protein / enzyme biologic “recombinant human acid alpha-glucosidase (rhGAA)” NCT00051935 ↗
Route Intravenous “20 mg/kg (qow); intravenous” NCT00051935 ↗
Target GAA “recombinant human GAA (rhGAA)” PMID 19775921 ↗ Jan 2010