Drugs / Olipudase alfa
Olipudase alfa
also known as
GZ402665 · Recombinant Human Acid Sphingomyelinase · rhASM · Olipudasa alfa · Olipudase alfa rpcp · Xenpozyme
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed chronic neurovisceral acid sphingomyelinase deficiency 3 milestones | |||||
| Filed | US (FDA) | — | chronic neurovisceral acid sphingomyelinase deficiency | 2022-02-04 | “A Biologics License Application (BLA) for olipudase alfa was submitted to the FDA, and the FDA designated the BLA for Priority Review.” sanofi.com ↗ “The FDA accepted for Priority Review the Biologics License Application (BLA) for olipudase alfa” sanofi.com ↗ |
| Filed | EU (EMA) | — | chronic neurovisceral acid sphingomyelinase deficiency | 2021-12-06 | “The European Medicines Agency (EMA) has accepted for review under an accelerated assessment procedure the Marketing Authorization Application (MAA) for olipudase alfa” sanofi.com ↗ |
| Filed | Japan (PMDA) | — | chronic neurovisceral acid sphingomyelinase deficiency | 2021-09-30 | “Olipudase alfa , an investigational recombinant human acid sphingomyelinase for the treatment of Acid Sphingomyelinase Deficiency (ASMD), was submitted in Japan” sanofi.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | EU (EMA) | Xenpozyme | — | 2022-06-24 | europa.eu ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT01722526 | Mar 2013 → Jan 2014 | acid sphingomyelinase deficiency | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT00410566 | Dec 2006 → Mar 2009 | acid sphingomyelinase deficiency | Genzyme, a Sanofi Company | Terminated | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02292654 | May 2015 → Dec 2019 | sphingolipidosis | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT06949358 | Nov 2021 → Apr 2025 | acid sphingomyelinase deficiency | Sanofi | Completed | No outcome recorded |
| Phase 2 | NCT02004704 | Dec 2013 → Sep 2023 | acid sphingomyelinase deficiency | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT02004691 | Dec 2015 → Mar 2021 | acid sphingomyelinase deficiency | Genzyme, a Sanofi Company | Completed | Mixed |
| Phase not stated1 trial | ||||||
| — | NCT04877132 | — | sphingolipidosis | Sanofi | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 9
| Date | Issuer | Release |
|---|---|---|
| 2023-09-14 | Sanofi | Results Media Update: New data at ERS showed Xenpozyme® (olipudase alfa) improved respiratory functions in adults with ASMD sanofi.com ↗ |
| 2022-08-31 | Sanofi | Regulatory Press Release: XenpozymeTM (olipudase alfa-rpcp) approved by FDA as first disease-specific treatment for ASMD (non-CNS manifestations) sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has approved Xenpozyme TM (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. |
| 2022-06-28 | Sanofi | Regulatory Press Release: Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD sanofi.com ↗
The European Commission (EC) has approved Xenpozyme ® (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B. |
| 2022-05-20 | Sanofi | Regulatory Media Update: CHMP recommends approval of Xenpozyme® (olipudase alfa), the first and only treatment for ASMD sanofi.com ↗
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Xenpozyme ® (olipudase alfa), recommending that this investigational enzyme replacement therapy be approved in the European Union (EU) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B. |
| 2022-03-28 | Sanofi | Regulatory Press Release: Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency sanofi.com ↗
The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Xenpozyme ® (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare, progressive, and potentially life-threatening genetic disease. |
| 2022-02-09 | Sanofi | Results Olipudase alfa shown to provide sustained improvement across multiple clinical manifestations of ASMD sanofi.com ↗ |
| 2021-12-06 | Sanofi | Regulatory EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD sanofi.com ↗
The European Medicines Agency (EMA) has accepted for review under an accelerated assessment procedure the Marketing Authorization Application (MAA) for olipudase alfa, Sanofi’s investigational enzyme replacement therapy which is being evaluated for the treatment of acid sphingomyelinase deficiency (ASMD). |
| 2021-02-02 | Sanofi | Results Media Update: Data presented at WORLDSymposiumTM reinforces robust rare disease pipeline and highlights additional clinical data for investigational avalglucosidase alfa in Pompe disease sanofi.com ↗
Adults with chronic sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase alfa: 1-year results of the ASCEND placebo-controlled trial |
| 2020-01-30 | Sanofi | Results Sanofi : Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomyelinase deficiency sanofi.com ↗
Olipudase alfa, an investigational recombinant human acid sphingomyelinase, demonstrated positive results in two separate clinical trials evaluating olipudase alfa for the treatment of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. |
All press releases naming this drug 10 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | olipudase alfa | “Olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is an investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD; Niemann-Pick...” PMID 26049896 ↗ May 2015 |
| Known as | GZ402665 | “olipudase alfa (GZ402665)” NCT04877132 ↗ |
| Known as | Olipudasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707358 ↗ |
| Known as | Olipudase alfa rpcp | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707358 ↗ |
| Known as | Recombinant Human Acid Sphingomyelinase | “Olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is an investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD; Niemann-Pick...” PMID 26049896 ↗ May 20151“A Phase I, Single-Center, Single Dose, Dose Escalation Study of Recombinant Human Acid Sphingomyelinase (rhASM) in Adults With Acid Sphingomyelinase Deficiency (ASMD)” NCT00410566 ↗ |
| Known as | rhASM | “Olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is an investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD; Niemann-Pick...” PMID 26049896 ↗ May 2015 |
| Known as | Xenpozyme | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707358 ↗ |
| Modality | Protein / enzyme biologic | “Recombinant Human Acid Sphingomyelinase” NCT00410566 ↗ |
| Route | Intravenous | “Single dose of 0.03mg/kg body weight IV” NCT00410566 ↗ |