Drugs / laronidase

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 · a red edge is where a trial was stopped

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
Also used as a comparator or background therapy in 3 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 32 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 41 trial
Phase 4 NCT00144781 Comparator Dec 2004 → Jan 2006 Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome Genzyme, a Sanofi Company Completed No outcome recorded

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

NCT00638547

NCT00176891

NCT00786968

NCT06406153

NCT00741338

NCT05134571

NCT00852358

NCT01173016

NCT00215527

NCT00144768

NCT00418821

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

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

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

NCT00144781

NCT00146757

NCT00146770

NCT04532047

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