Drugs / Avalglucosidase alfa
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed glycogen storage disease II 2 milestones | |||||
| Filed | US (FDA) | — | glycogen storage disease II | 2020-11-18 | “In November 2020, the U.S. Food and Drug Administration (FDA) accepted for Priority Review the Biologics License Application (BLA) for avalglucosidase alfa” sanofi.com ↗ “The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa” sanofi.com ↗ “The FDA accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa” sanofi.com ↗ |
| Filed | EU (EMA) | — | glycogen storage disease II | 2020-10-02 | “In October 2020, the European Medicines Agency accepted for review the Marketing Authorization Application for avalglucosidase alfa” sanofi.com ↗ “The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa” sanofi.com ↗ “In October, the European Medicines Agency accepted for review the Marketing Authorization Application for avalglucosidase alfa” sanofi.com ↗ “The EMA accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa” sanofi.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Nexviadyme | — | 2022-06-24 | europa.eu ↗ |
| Filed | Japan (PMDA) | — | — | — | “Sanofi also filed avalglucosidase alfa in Japan in January 2021.” sanofi.com ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT01898364 | Aug 2013 → Feb 2015 | glycogen storage disease II | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 23 trials | ||||||
| Phase 2 | NCT03019406 | Oct 2017 → Sep 2019 overdue | glycogen storage disease due to acid maltase deficiency, infantile onset | Genzyme, a Sanofi Company | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02032524 | Feb 2014 → Dec 2022 | glycogen storage disease II | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 2 | NCT00025896 | May 2001 → Sep 2002 | glycogen storage disease due to acid maltase deficiency, infantile onset | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 32 trials · 2 met primary | ||||||
| Phase 3 | NCT04910776 | Sep 2021 → Mar 2026 overdue | glycogen storage disease II | Sanofi | Active not recruiting | Met primary |
| Phase 3 | NCT02782741 | Nov 2016 → Mar 2020 | glycogen storage disease due to acid maltase deficiency, late-onset | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase 43 trials | ||||||
| Phase 4 | NCT06666413 | May 2025 → May 2028 expected | glycogen storage disease II | Genzyme, a Sanofi Company | Recruiting | No outcome recorded |
| Phase 4 | NCT07652814 | Oct 2022 → Mar 2028 expected | glycogen storage disease due to acid maltase deficiency, late-onset | Iris Plug | Active not recruiting | No outcome recorded |
| Phase 4 | NCT05164055 | Jul 2022 → Jun 2027 expected | glycogen storage disease II | Genzyme, a Sanofi Company | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2026-06-30 | Sanofi | Results Press Release: Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study sanofi.com ↗
Nexviazyme met its primary endpoint, participants alive and free of invasive ventilation, in treatment-naïve infants zero to six months of age in the Baby-COMET phase 3 study |
| 2024-02-05 | Sanofi | Results Media Update: New data demonstrate clinical safety, benefit and durability of Nexviazyme® across a wide-range of Pompe disease patient groups sanofi.com ↗
New data suggest treatment with Nexviazyme ® (avalglucosidase alfa) meaningfully improved ptosis, or drooping eyelid, in pediatric patients with infantile-onset Pompe disease (IOPD) over nearly three years. |
| 2023-10-06 | Sanofi | Results Media Update: New data at WMS 2023 reaffirm long-term efficacy of Nexviazyme® (avalglucosidase alfa) for the treatment of Pompe disease sanofi.com ↗ |
| 2023-02-24 | Sanofi | Results Press release: New Phase 3 data presented at WORLDSymposium™ reinforce Nexviazyme® (avalglucosidase alfa) as potential new standard of care for all people living with late-onset Pompe disease sanofi.com ↗
Additionally, a separate analysis of respiratory function showed clinical benefit over two years for patients who switched to Nexviazyme regardless of prior response to alglucosidase alfa. |
| 2022-06-28 | Sanofi | Regulatory Press Release: Nexviadyme® (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe Disease sanofi.com ↗
The European Commission has granted marketing authorization for Nexviadyme ® (avalglucosidase alfa), an enzyme replacement therapy (ERT) for the long-term treatment of both late-onset and infantile-onset Pompe disease, a rare, progressive and debilitating muscle disorder. |
| 2022-02-08 | Sanofi | Results Nexviazyme® (avalglucosidase alfa) shows sustained improvements in respiratory function and mobility in patients with Pompe disease sanofi.com ↗
New data demonstrate people living with late-onset Pompe disease (LOPD), a rare muscle disorder, maintained improvements in respiratory function and mobility following nearly two years of treatment with Nexviazyme ® (avalglucosidase alfa). |
| 2021-11-12 | Sanofi | Regulatory Sanofi announces results of CHMP re-examination of the New Active Substance status for avalglucosidase alfa, a potential new standard of care for the treatment of Pompe disease sanofi.com ↗
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) today reaffirmed its opinion that avalglucosidase alfa does not qualify as a New Active Substance (NAS). |
| 2021-08-06 | Sanofi | Regulatory FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has approved Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe. |
| 2021-07-27 | Sanofi | Regulatory Sanofi provides update on avalglucosidase alfa EU submission for patients with Pompe Disease sanofi.com ↗
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa, a long-term enzyme replacement therapy for the treatment of people with Pompe disease. |
| 2020-11-18 | Sanofi | Regulatory FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). |
| 2020-10-02 | Sanofi | Regulatory EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease sanofi.com ↗
The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). |
| 2020-06-16 | Sanofi | Results Sanofi’s investigational enzyme replacement therapy shows clinically meaningful improvement in critical manifestations of late-onset Pompe disease sanofi.com ↗
Avalglucosidase alfa met the primary endpoint demonstrating non-inferiority in improving respiratory function compared to alglucosidase alfa (standard of care) in patients with LOPD. |
| 2020-06-08 | Sanofi | Regulatory Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to avalglucosidase alfa for the treatment of patients with a confirmed diagnosis of Pompe disease. |
All press releases naming this drug 14 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Avalglucosidase alfa | “Avalglucosidase alfa, a recombinant human GAA enzyme replacement therapy designed for increased cellular uptake and glycogen clearance, has been studied for long-term efficacy...” PMID 35618441 ↗ Aug 2022 |
| Known as | GZ402666 | “A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA,...” NCT02782741 ↗ |
| Known as | neoGAA | “A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA,...” NCT02782741 ↗ |
| Known as | recombinant human acid alpha-glucosidase | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00025896 ↗ |
| Known as | rhGAA | “recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease.” NCT00025896 ↗ |
| Modality | Protein / enzyme biologic | “Enzyme replacement therapy with recombinant human GAA (rhGAA)” PMID 19775921 ↗ Jan 2010 |
| Route | Intravenous | “treated with IV rhGAA” PMID 19775921 ↗ Jan 2010 |
| Target | GAA | “Avalglucosidase alfa, a recombinant human GAA enzyme replacement therapy designed for increased cellular uptake and glycogen clearance” PMID 35618441 ↗ Aug 2022 |