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

Milestone	Jurisdiction	Brand	Indication	Date	Sentence 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 · a red edge is where a trial was stopped

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 NCT00765414 · Ph 2 NCT00059280 · Ph 2/3 NCT00763932 · Ph 2 NCT00053573 · Ph 1/2 NCT00051935 · Ph 2

2003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
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	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	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	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
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	No outcome recorded
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

Also used as a comparator or background therapy in 7 trials

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 1/21 trial
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
Phase 21 trial
Phase 2	NCT01380743 Background	Oct 2011 → Jan 2013	glycogen storage disease II	Amicus Therapeutics	Completed	No outcome recorded
Phase 33 trials
Phase 3	NCT03729362 Comparator	Dec 2018 → Dec 2020	glycogen storage disease due to acid maltase deficiency, late-onset	Amicus Therapeutics	Completed	No outcome recorded
Phase 3	NCT02782741 Comparator	Nov 2016 → Mar 2020	glycogen storage disease due to acid maltase deficiency, late-onset	Genzyme, a Sanofi Company	Completed	No outcome recorded
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
Phase 42 trials
Phase 4	NCT01597596 Comparator	Aug 2012 → Dec 2014	glycogen storage disease due to acid maltase deficiency, infantile onset	Genzyme, a Sanofi Company	Terminated	No outcome recorded
Phase 4	NCT00701701 Background	Dec 2008 → Feb 2020	glycogen storage disease II	Genzyme, a Sanofi Company	Terminated	No outcome recorded

Evidence & citations 22 cited values

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

Field	Value	Cited 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 ↗ NCT03729362 ↗ NCT00158600 ↗ NCT03687333 ↗ NCT00486889 ↗ NCT04676373 ↗ NCT00520143 ↗ NCT00051935 ↗ NCT01597596 ↗ NCT00701129 ↗ NCT01288027 ↗ NCT00455195 ↗ NCT00074919 ↗ NCT03019406 ↗ NCT01526785 ↗ NCT00701701 ↗ NCT02782741 ↗ NCT00483379 ↗
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 ↗ NCT00765414 ↗ NCT00125879 ↗ NCT00268944 ↗ NCT00763932 ↗ NCT00250939 ↗ NCT00059280 ↗ NCT00074932 ↗
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