Drugs / elosulfase alfa
elosulfase alfa
also known as
BMN 110 · Bmn 110, rhgalns, n-acetylgalactosamine-6-sulfatase, chondroitinsulfatase, galactose-6-sulfate sulfatase, ec=3.1.6.4 · Chondroitin sulfatase · Chondroitinase · Chondrosulfatase · E.c. 3.1.6.4 · Elosulfasa alfa · Recombinant human n-acetylgalactosamine-6-sulfatase · +3 more
Protein / enzyme biologic targets GALNS
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed mucopolysaccharidosis, type 10 2 milestones | |||||
| Filed | EU (EMA) | Vimizim | mucopolysaccharidosis, type 10 | 2013-04-24 | “submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Vimizim (BMN-110, elosulfase alfa)” biomarin.com ↗ |
| Filed | US (FDA) | Vimizim | mucopolysaccharidosis, type 10 | 2013-04-01 | “submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Vimizim (BMN-110, elosulfase alfa)” biomarin.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Vimizim | — | 2014-04-27 | europa.eu ↗ |
| Approved | US (FDA) | VIMIZIM | — | 2014-02-14 | fda.gov ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT01242111 | Nov 2010 → Jul 2014 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 1/2 | NCT00884949 | Apr 2009 → Feb 2011 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 23 trials | ||||||
| Phase 2 | NCT01697319 | Aug 2012 → Oct 2014 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 2 | NCT01609062 | Apr 2012 → Nov 2014 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 2 | NCT01515956 | Oct 2011 → Feb 2016 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 33 trials | ||||||
| Phase 3 | NCT01966029 | Jul 2013 → Jul 2016 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT01415427 | Jul 2011 → Jun 2016 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT01275066 | Feb 2011 → Aug 2012 | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Completed | Mixed |
| Phase not stated1 trial | ||||||
| — | NCT01858103 | — | mucopolysaccharidosis type 4A | BioMarin Pharmaceutical | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 19
| Date | Issuer | Release |
|---|---|---|
| 2019-06-04 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Approval of Vimizim® (elosulfase alfa) in China for Treatment of Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that Vimizim® (elosulfase alfa) has been approved by the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. |
| 2017-02-15 | BioMarin Pharmaceutical | Results BioMarin Announces 11 Presentations at 13th Annual WORLDSymposium™ 2017 biomarin.com ↗
Elosulfase alfa treatment and changes in physical functioning and disability in Morquio syndrome type A |
| 2015-11-23 | BioMarin Pharmaceutical | Regulatory NICE Recommends BioMarin’s Vimizim® (elosulfase alfa) for the Treatment of Morquio A Syndrome in England biomarin.com ↗ |
| 2014-12-29 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Approval of VIMIZIM(R) (elosulfase alfa) in Japan for Treatment of Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the Ministry of Health, Labor and Welfare granted approval of the registration of VIMIZIM® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. |
| 2014-12-10 | BioMarin Pharmaceutical | Regulatory BioMarin’s VIMIZIM(R) (elosulfase alfa) Approved in Brazil for Treatment of Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Agência Nacional de Vigilancia Sanitaria (ANVISA), or the National Health Surveillance Agency Brazil, has approved VIMIZIM® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. |
| 2014-12-07 | BioMarin Pharmaceutical | Regulatory BioMarin’s VIMIZIM(R) (elosulfase alfa) Approved in Australia for Treatment of Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the Australian Therapeutic Goods Administration (TGA) granted approval of the registration of VIMIZIM® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. |
| 2014-07-07 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Health Canada Approval of VIMIZIM™ (elosulfase alfa) for the Treatment of Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that Health Canada has approved VIMIZIM™(elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. |
| 2014-04-28 | BioMarin Pharmaceutical | Regulatory BioMarin Announces European Commission Approval for VIMIZIM(R) (elosulfase alfa) for the Treatment of Morquio A Syndrome in Patients of All Ages biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Commission has granted marketing authorization for VIMIZIM® (elosulfase alfa), the first specific treatment approved in the European Union for Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) in patients of all ages. |
| 2014-02-20 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Positive Opinion From the CHMP in the European Union for VIMIZIM(TM) (elosulfase alfa) for Morquio A Syndrome biomarin.com ↗
The CHMP's recommendation is now referred to the European Commission (EC). |
| 2014-02-17 | BioMarin Pharmaceutical | Regulatory BioMarin to Discuss FDA Approval of VIMIZIM(TM) and Provide Preliminary 2013 Results biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) will host a conference call and webcast on Tuesday, February 18, at 8:00 a.m. ET to discuss FDA approval of VIMIZIM, (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). |
| 2014-02-14 | BioMarin Pharmaceutical | Regulatory BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved VIMIZIM™ (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). |
| 2013-11-15 | BioMarin Pharmaceutical | Regulatory FDA Posts Briefing Documents for Advisory Committee Meeting Reviewing Vimizim(TM) for the Treatment of Morquio A Syndrome biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the U.S. Food and Drug Administration (FDA) posted briefing materials for the November 19, 2013 Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting to review the Vimizim™ (elosulfase alfa) Biologics License Application (BLA). |
| 2013-07-01 | BioMarin Pharmaceutical | Regulatory Vimizim Marketing Application Submitted to ANVISA in Brazil biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that it has submitted the Vimizim Marketing Application (MA) to the Agência Nacional de Vigilancia Sanitaria (ANVISA), or the National Health Surveillance Agency Brazil. |
| 2013-05-30 | BioMarin Pharmaceutical | Regulatory FDA Accepts Vimizim BLA and Grants Priority Review Designation biomarin.com ↗
The FDA has granted priority review designation to Vimizim, which is granted to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. |
| 2013-04-24 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Vimizim MAA to EMA for the Treatment of MPS IVA biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Vimizim (BMN-110, elosulfase alfa), |
| 2013-04-01 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Vimizim BLA to the U.S. FDA for the Treatment of MPS IVA biomarin.com ↗ |
| 2010-04-29 | BioMarin Pharmaceutical | Results BioMarin Reports Positive Results for Phase I/II Trial for BMN 110 for MPS IVA biomarin.com ↗
BioMarin Pharmaceutical Inc. today announced positive results for the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. |
| 2010-02-04 | BioMarin Pharmaceutical | Results BioMarin Reports Encouraging Preliminary Data on BMN 110 for MPS IVA biomarin.com ↗
Keratan sulfate (KS) levels fall within a few weeks after the start of therapy. |
| 2009-03-18 | BioMarin Pharmaceutical | Regulatory BioMarin’s Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that its application for clinical trial authorization (CTA) for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome, has been accepted by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA). |
All press releases naming this drug 24 releases
Evidence & citations 15 cited values
Every value below carries the sentence it was read from. 10 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | elosulfase alfa | “BACKGROUND: Long-term safety and efficacy of elosulfase alfa enzyme replacement therapy (ERT) were assessed in 173 patients with Morquio A syndrome (mucopolysaccharidosis IVA)...” PMID 29248359 ↗ Dec 20171“Elosulfase alfa is an enzyme replacement therapy that provides a treatment option for patients with Morquio A.” PMID 25487082 ↗ Dec 2014 |
| Known as | BMN 110 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01609062 ↗ |
| Known as | Bmn 110, rhgalns, n-acetylgalactosamine-6-sulfatase, chondroitinsulfatase, galactose-6-sulfate sulfatase, ec=3.1.6.4 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Chondroitin sulfatase | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Chondroitinase | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Chondrosulfatase | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | E.c. 3.1.6.4 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Elosulfasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Recombinant human n-acetylgalactosamine-6-sulfatase | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Recombinant human n-acetylgalactosamine-6-sulfatase (rhgalns) | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | RHGALNS | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Known as | Vimizim | ChEMBL registry synonym — accepted as the source's own label CHEMBL2108676 ↗ |
| Modality | Protein / enzyme biologic | “Elosulfase alfa is an enzyme replacement therapy” PMID 25487082 ↗ Dec 2014 |
| Route | Intravenous | “Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110” NCT00884949 ↗ |
| Target | GALNS | “Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS” NCT01966029 ↗ |