Drugs / Galsulfase
Galsulfase
also known as
N-acetylgalactosamine 4-sulfatase · Naglazyme · rhASB · Argyplase · Arylsulfatase b galsulfase · Aryplase · Galsulfasa · Neglazyme
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed mucopolysaccharidosis type 6 3 milestones | |||||
| Filed | Japan (PMDA) | Naglazyme | mucopolysaccharidosis type 6 | 2007-08-13 | “has submitted a Biologics License Application (BLA) for Naglazyme(R) (galsulfase) to the Japanese Ministry of Health, Labour and Welfare.” biomarin.com ↗ |
| Filed | EU (EMA) | Aryplase | mucopolysaccharidosis type 6 | 2004-12-05 | “it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for Aryplase(TM) (galsulfase), an investigational enzyme” biomarin.com ↗ |
| Filed | US (FDA) | Aryplase | mucopolysaccharidosis type 6 | 2004-11-28 | “it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Aryplase(TM) (galsulfase)” biomarin.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | NAGLAZYME | — | 2011-04-28 | fda.gov ↗ |
| Approved | EU (EMA) | Naglazyme | — | 2006-01-23 | europa.eu ↗ |
| Approved | US (FDA) | NAGLAZYME | — | 2005-05-31 | fda.gov ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT00048620 | Sep 2000 → Nov 2005 | mucopolysaccharidosis type 6 | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT00048711 | Mar 2002 → Jun 2006 | mucopolysaccharidosis type 6 | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT00104234 | Feb 2004 → Oct 2006 | mucopolysaccharidosis type 6 | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT00299000 | May 2006 → Apr 2009 | mucopolysaccharidosis type 6 | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT02156674 | Jan 2016 → Dec 2019 | mucopolysaccharidosis type 6 | Masonic Cancer Center, University of Minnesota | Terminated | No outcome recorded Stop: Enrollment |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT00067470 Comparator | Sep 2003 → Mar 2004 | mucopolysaccharidosis type 6 | BioMarin Pharmaceutical | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2017-02-15 | BioMarin Pharmaceutical | Results BioMarin Announces 11 Presentations at 13th Annual WORLDSymposium™ 2017 biomarin.com ↗
Long-term galsulfase treatment improves survival of patients with mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome): 15 year follow up from the survey study |
| 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 ↗
The data suggests that patients treated with Naglazyme lived longer, had long-term improvement in endurance, and experienced improvement in pulmonary function and growth. |
| 2011-07-28 | BioMarin Pharmaceutical | Results BioMarin Provides Update on GALNS Phase 1/2 Extension Study (MOR-100) biomarin.com ↗
Looking at the Phase 1/2 data in totality, we now see that the endurance and pulmonary function improvements are durable and clinically meaningful compared to baseline, and similar in magnitude to what we have observed during the development of Naglazyme and Aldurazyme. |
| 2009-02-03 | BioMarin Pharmaceutical | Regulatory Naglazyme Approved by Brazil’s National Health Surveillance Agency biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that ANVISA, Brazil's National Health Surveillance Agency, has granted BioMarin marketing approval for Naglazyme(R) (galsulfase) for the treatment of patients with Mucopolysaccharidosis VI (MPS VI). |
| 2008-03-31 | BioMarin Pharmaceutical | Regulatory Naglazyme Approved by Japanese Ministry of Health biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that AnGes MG, Inc. (AnGes), BioMarin's marketing and distribution partner in Japan, has received approval for its Marketing Application for Naglazyme(R) (galsulfase) from the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with Mucopolysaccharidosis VI ( MPS VI). |
| 2007-08-13 | BioMarin Pharmaceutical | Regulatory BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that AnGes MG, Inc. (AnGes), BioMarin's marketing and distribution partner in Japan, has submitted a Biologics License Application (BLA) for Naglazyme(R) (galsulfase) to the Japanese Ministry of Health, Labour and Welfare. |
| 2006-01-30 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Marketing Approval for Naglazyme in European Union biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the European Commission has granted marketing authorization for Naglazyme(TM) (galsulfase), the first specific treatment approved in the European Union for patients with the genetic disease mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). |
| 2005-09-15 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Positive Opinion for Naglazyme From European Regulatory Authority biomarin.com ↗
The positive opinion from the CHMP is the final step before formal approval to market Naglazyme in the 25 member states of the European Union, Iceland and Norway. |
| 2005-05-31 | BioMarin Pharmaceutical | Regulatory BioMarin Announces FDA Approval for Naglazyme biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Naglazyme(TM) (galsulfase), the first specific therapy approved for the treatment of mucopolysaccharidosis VI (MPS VI). |
| 2005-03-08 | BioMarin Pharmaceutical | Results BioMarin’s Phase 3 Extension Study of rhASB in MPS VI Demonstrates Additional Improvements in Endurance biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced positive results from the Phase 3 extension study of rhASB (galsulfase), the company's investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). |
| 2005-01-31 | BioMarin Pharmaceutical | Regulatory FDA Accepts and Grants Six-Month Review for BioMarin’s Marketing Application for rhASB for MPS VI biomarin.com ↗
The FDA will take action on the application, under the Prescription Drug User Fee Act (PDUFA), by May 31, 2005. |
| 2004-12-05 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Marketing Authorization Application in European Union for Aryplase to Treat MPS VI biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for Aryplase(TM) (galsulfase), an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). |
| 2004-11-28 | BioMarin Pharmaceutical | Regulatory BioMarin Submits U.S. Biologics License Application for Marketing Authorization of Aryplase for MPS VI biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Aryplase(TM) (galsulfase), an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). |
| 2004-06-02 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Phase 3 Data on Aryplase(TM) for MPS VI biomarin.com ↗
BioMarin Pharmaceutical Inc. today announced positive results from its Phase 3 clinical trial of Aryplase(TM), an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). |
All press releases naming this drug 20 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Galsulfase | “Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI.” PMID 24108527 ↗ Oct 2013 |
| Known as | Argyplase | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201822 ↗ |
| Known as | Arylsulfatase b galsulfase | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201822 ↗ |
| Known as | Aryplase | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201822 ↗ |
| Known as | Galsulfasa | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201822 ↗ |
| Known as | N-acetylgalactosamine 4-sulfatase | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00104234 ↗ |
| Known as | Naglazyme | “Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI.” PMID 24108527 ↗ Oct 2013 |
| Known as | Neglazyme | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201822 ↗ |
| Known as | rhASB | “weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB)” NCT00048620 ↗1“1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB)” NCT00048711 ↗ |
| Modality | Protein / enzyme biologic | “recombinant human N-acetylgalactosamine 4-sulfatase” NCT00048620 ↗ |
| Route | Intravenous | “weekly intravenous infusions” NCT00048620 ↗ |