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Drugs / laronidase
last change Oct 2025 re-read 9 days ago

laronidase

also known as Aldurazyme · Recombinant Human Alpha-L-Iduronidase · A-l-iduronidase · Alronidase · Ec 3.2.1.76 · Laronidasa · rhIDU · YW17

Protein / enzyme biologic

Developed for
Hurler syndrome · Hurler-Scheie syndrome · mucopolysaccharidosis type 1 · Scheie syndrome · cognitive disorder · spinal cord disorder · Gaucher disease type II · Gaucher disease type III
+6 more · glycogen storage disease due to acid maltase deficiency, infantile onset · mucopolysaccharidosis type 2 · mucopolysaccharidosis type 4A · mucopolysaccharidosis type 6 · mucopolysaccharidosis type 7 · Wolman disease
Investigated by
Genzyme, a Sanofi Company · BioMarin/Genzyme LLC · The Ryan Foundation · Masonic Cancer Center, University of Minnesota · Patricia I. Dickson, M.D. · University of California, Los Angeles
+12 more · National Institute of Neurological Disorders and Stroke (NINDS) · Rare Diseases Clinical Research Network · University of Minnesota · BioMarin Pharmaceutical · Cinnagen · Duke University · FDA Office of Orphan Products Development · National Center for Advancing Translational Sciences (NCATS) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · UCSF Benioff Children's Hospital Oakland · University of California, San Francisco · agnes chen

Regulatory milestones approvals, filings & regulatory actions · 3 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) ALDURAZYME — 2009-05-20 fda.gov ↗
Approved EU (EMA) Aldurazyme — 2003-06-09 europa.eu ↗
Approved US (FDA) ALDURAZYME — 2003-04-30 fda.gov ↗

Trials 15

today
US approved 2003
EU approved 2003
NCT06406153 · Ph 3 NCT05134571 · Ph 4 NCT04532047 · Ph 1 NCT02232477 · not/applicable NCT01173016 · Ph 1 NCT00418821 · Ph 4 NCT00852358 · not/applicable NCT00741338 · Ph 1/2 NCT00786968 · Ph 1 NCT00638547 · Ph 1 NCT00258011 · Ph 3 NCT00215527 · Ph 1 NCT00144768 · Ph 4 NCT00176891 · Ph 2 NCT00146757 · Ph 2
2002200320042005200620072008200920102011201220132014201520162017201820192020202120222023202420252026202720282029203020312032
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 15 trials
Phase 1 NCT04532047 Jul 2021 → Jul 2031 expected Gaucher disease type II, Gaucher disease type III, Wolman disease, glycogen storage disease due to acid maltase deficiency, infantile onset +5 University of California, San Francisco Recruiting No outcome recorded
Phase 1 NCT01173016 May 2012 → Mar 2016 Hurler syndrome Masonic Cancer Center, University of Minnesota Completed No outcome recorded
Phase 1 NCT00638547 Jan 2008 → Feb 2017 Hurler syndrome Masonic Cancer Center, University of Minnesota Completed No outcome recorded
Phase 1 NCT00786968 Jan 2008 → Oct 2011 Hurler-Scheie syndrome, Scheie syndrome, spinal cord disorder Patricia I. Dickson, M.D. Terminated No outcome recorded
Phase 1 NCT00215527 Nov 2005 → Oct 2011 mucopolysaccharidosis type 1, spinal cord disorder Patricia I. Dickson, M.D. Terminated No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT00741338 Sep 2008 → Sep 2012 mucopolysaccharidosis type 1 Genzyme, a Sanofi Company Completed No outcome recorded
Phase 22 trials
Phase 2 NCT00176891 Mar 2004 → Nov 2012 Hurler syndrome Masonic Cancer Center, University of Minnesota Completed No outcome recorded
Phase 2 NCT00146757 Oct 2002 → May 2005 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded
Phase 32 trials
Phase 3 NCT06406153 Sep 2022 → Aug 2023 mucopolysaccharidosis type 1 Cinnagen Completed No outcome recorded
Phase 3 NCT00258011 Dec 2005 → Oct 2006 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded
Phase 43 trials
Phase 4 NCT05134571 Oct 2021 → Jul 2023 mucopolysaccharidosis type 1 Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT00418821 Oct 2010 → Dec 2022 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Terminated No outcome recorded
Phase 4 NCT00144768 Jul 2004 → May 2007 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded
Phase not applicable2 trials
— NCT02232477 Aug 2014 → Feb 2020 cognitive disorder, mucopolysaccharidosis type 1 agnes chen Terminated No outcome recorded
— NCT00852358 Jun 2009 → Apr 2015 Hurler-Scheie syndrome, Scheie syndrome, cognitive disorder Patricia I. Dickson, M.D. Completed No outcome recorded
Comparator or background therapy3 trials
Phase 3 NCT00146770 Comparator May 2001 → Mar 2005 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded
Phase 3 NCT00912925 Comparator Dec 2000 → Sep 2001 Hurler syndrome, Hurler-Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded
Phase 4 NCT00144781 Comparator Dec 2004 → Jan 2006 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded

News releases announcing trial results or a regulatory action · 3

DateIssuerRelease
2006-10-31 BioMarin Pharmaceutical Regulatory BioMarin Announces Marketing Approval for Aldurazyme in Japan biomarin.com ↗
Aldurazyme was approved in Japan as an orphan drug.
2003-12-29 BioMarin Pharmaceutical Results PNAS Study from BioMarin Highlights Possible New Approach for Improving the Efficacy of Enzyme and Protein Replacement Therapies biomarin.com ↗
The induction of long-term immune tolerance required both components of the regimen: the high-uptake rhIDU and the immunosuppressive agents
2003-09-03 BioMarin Pharmaceutical Results BioMarin Demonstrates Normalization of Carbohydrate Storage in Brain Tissue Using an MPS I Model biomarin.com ↗
BioMarin Pharmaceutical Inc. announced results from studies indicating that intrathecal injection of recombinant human alpha-L-iduronidase (rhIDU) can reduce carbohydrate storage in brain tissue in the canine model of MPS I (mucopolysaccharidosis I).

All press releases naming this drug 7 releases

DateIssuerRelease

Evidence & citations 12 cited values

Every value below carries the sentence it was read from. 21 sources stand behind the page.

FieldValueCited text
Known as laronidase “Our goal was to evaluate the long-term safety and efficacy of recombinant human alpha-l-iduronidase (laronidase) in patients with mucopolysaccharidosis I.” PMID 19117887 ↗ Jan 2009
16

“evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease” NCT00258011 ↗

“A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease” NCT00144781 ↗

“laronidase 1.74 mg IT q 3 months” NCT02232477 ↗

“Aldurazyme® (laronidase)” NCT00146757 ↗

“Aldurazyme (laronidase)” NCT04532047 ↗

NCT00741338 ↗

NCT05134571 ↗

NCT01173016 ↗

NCT00638547 ↗

NCT06406153 ↗

Show 6 more sources
Known as A-l-iduronidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201595 ↗
Known as Aldurazyme “Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over...” NCT00912925 ↗
8

“Laronidase (Aldurazyme®) stands as the sole FDA-approved enzyme replacement therapy (ERT) for MPS I to date.” PMID 41163043 ↗ Oct 2025

“Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.” NCT06406153 ↗

“Recombinant Human Alpha-L-Iduronidase, Aldurazyme®” NCT00144768 ↗

“Aldurazyme (Recombinant Human Alpha-L-Iduronidase)” NCT00258011 ↗

NCT04532047 ↗

NCT00144781 ↗

NCT00146770 ↗

NCT00146757 ↗
Known as Alronidase ChEMBL registry synonym — accepted as the source's own label CHEMBL1201595 ↗
Known as Ec 3.2.1.76 ChEMBL registry synonym — accepted as the source's own label CHEMBL1201595 ↗
Known as Laronidasa ChEMBL registry synonym — accepted as the source's own label CHEMBL1201595 ↗
Known as Recombinant Human Alpha-L-Iduronidase “Patients received Aldurazyme (recombinant human alpha-L-iduronidase (rhIDU)) once per week” NCT00146757 ↗
1

“Recombinant Human Alpha-L-Iduronidase, Aldurazyme®” NCT00144768 ↗
Known as rhIDU “Patients received Aldurazyme (recombinant human alpha-L-iduronidase (rhIDU)) once per week” NCT00146757 ↗
Known as YW17 “Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.” NCT06406153 ↗
Action Restore “Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup.” NCT00638547 ↗
Modality Protein / enzyme biologic “Recombinant Human Alpha-L-Iduronidase, Aldurazyme®” NCT00144768 ↗
Route Intravenous “dose of 0.58mg/kg body weight IV every week” NCT00144768 ↗