Drugs / vestronidase alfa
vestronidase alfa
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | MEPSEVII | — | 2020-12-23 | fda.gov ↗ |
| Approved | EU (EMA) | Mepsevii | — | 2018-08-23 | europa.eu ↗ |
| Approved | US (FDA) | MEPSEVII | — | 2017-11-15 | fda.gov ↗ |
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01856218 | Nov 2013 → Jul 2016 | mucopolysaccharidosis type 7 | Ultragenyx Pharmaceutical Inc | Completed | No outcome recorded |
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT02418455 | Jul 2015 → Mar 2019 | mucopolysaccharidosis type 7 | Ultragenyx Pharmaceutical Inc | Completed | Met primary |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT02432144 | Nov 2015 → Jan 2019 | mucopolysaccharidosis type 7 | Ultragenyx Pharmaceutical Inc | Completed | No outcome recorded |
| Phase 3 | NCT02230566 | Dec 2014 → May 2016 | mucopolysaccharidosis type 7 | Ultragenyx Pharmaceutical Inc | Completed | Met primary |
| Phase not stated2 trials | ||||||
| — | NCT03775174 | — | mucopolysaccharidosis type 7 | Ultragenyx Pharmaceutical Inc | Available | No outcome recorded |
| — | NCT02097251 | — | mucopolysaccharidosis type 7 | Joyce Fox | No longer available | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2018-10-18 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Approval of Mepsevii™ (vestronidase alfa) in Brazil for the Treatment of Mucopolysaccharidosis VII ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Brazil’s National Health Surveillance Agency (ANVISA) has approved Mepsevii™ (vestronidase alfa) for the treatment of Mucopolysaccharidosis VII (MPS VII; Sly syndrome) for patients of all ages. |
| 2018-08-27 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Approval of Mepsevii™ (vestronidase alfa) in Europe for the Treatment of Mucopolysaccharidosis VII ultragenyx.com ↗
Mepsevii is now approved for use in all 28 EU countries and in Iceland , Liechtenstein and Norway . |
| 2018-06-29 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Positive CHMP Opinion for Mepsevii™ (vestronidase alfa) For the Treatment of Mucopolysaccharidosis VII ultragenyx.com ↗ |
| 2017-11-15 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces FDA Approval of MEPSEVII™ (vestronidase alfa), the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis VII ultragenyx.com ↗
The approval of MEPSEVII is a pivotal moment for Ultragenyx and for patients suffering from ultra-rare genetic diseases for which the investment and development of treatments has not happened yet, |
| 2017-05-23 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Recombinant Human Beta-Glucuronidase Biologics License Application and Marketing Authorization Application Filed and Accepted for Review; FDA Grants Priority Review Status ultragenyx.com ↗
The FDA granted rhGUS Priority Review status, which is available for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. |
| 2016-07-14 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Topline Data from Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7 ultragenyx.com ↗
The study met its primary endpoint of reducing urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment, demonstrating a reduction from baseline of 64.8 percent (p0.0001). |
| 2015-02-10 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive 36-Week Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7 ultragenyx.com ↗
The 36-week results showed a greater change in urinary GAG excretion at the higher 4 mg/kg dose of rhGUS, with a mean urinary GAG reduction of approximately 60%. |
| 2014-09-03 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Interim Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7 ultragenyx.com ↗
Results from the primary analysis phase show evidence of clearance of lysosomal storage as indicated by the decline in urinary glycosaminoglycan (GAG) excretion and the reduction in liver size. |
| 2014-03-27 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Preliminary Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7 ultragenyx.com ↗
Preliminary results from three patients who have been administered 2 mg/kg of rhGUS every other week for two, six, and 12 weeks show evidence of clearance of lysosomal storage as indicated by the decline in urinary glycosaminoglycan (GAG) excretion beginning at two weeks of treatment of approximately 30-50%. |
| 2014-02-12 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Presentation of Data From a Single Patient Treated With Recombinant Human Beta-Glucuronidase at 10th Annual World Lysosomal Disease Network Symposium ultragenyx.com ↗
Preliminary data showed a reduction in lysosomal storage based on reduced excretion of urinary glycosaminoglycans and a reduction in the size of the enlarged liver and spleen. |
| 2013-08-14 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial Testing UX003 in Mucopolysaccharidosis Type 7 (MPS 7) ultragenyx.com ↗
Ultragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial Testing UX003 in Mucopolysaccharidosis Type 7 (MPS 7) |
| 2012-03-28 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7 ultragenyx.com ↗
Ultragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7 |
| 2012-02-28 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7) ultragenyx.com ↗
Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7) |
All press releases naming this drug 14 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | vestronidase alfa | “The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.” PMID 32063397 ↗ Jan 20201“Vestronidase alfa, a recombinant human GUS, is an enzyme replacement therapy approved in the US and EU for the treatment of MPS VII.” PMID 30467742 ↗ May 2019 |
| Known as | Mepsevii | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03775174 ↗ |
| Known as | UX003 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02418455 ↗ |
| Known as | Vestronidasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707382 ↗ |
| Known as | Vestronidase alfa vjbk | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707382 ↗ |
| Modality | Protein / enzyme biologic | “Vestronidase alfa, a recombinant human GUS, is an enzyme replacement therapy” PMID 30467742 ↗ May 2019 |
| Route | Intravenous | “via intravenous (IV) administration every other week (QOW)” NCT01856218 ↗ |