Product Description
Pegunigalsidase alfa is designed to be a plant cell culture-expressed, and a chemically modified version of, the recombinant alpha-Galactosidase-A protein. Protein sub-units are covalently bound via chemical cross-linking using PEG chains, resulting in a more active and stable molecule compared to the current available versions of the molecule as seen in preclinical models. In clinical research, pegunigalsidase alfa appears to have a favorable circulatory half-life, with targeted enzyme activity in organs affected by Fabry disease. (Sourced from: https://protalix.com/products/pegunigalsidase-alfa/)
Mechanisms of Action: Enzyme Replacement Therapy, GLA
Novel Mechanism: No
Modality: Peptide/Protein
Route of Administration: Intravenous
FDA Designation: *
Approval Status: Approved
Approved Countries: Belgium | Croatia | Czech | European Medicines Agency | Finland | Hungary | Iceland | Ireland | Lithuania | Poland | Portugal | Slovakia | Sweden | United States
Approved Indications: None
Company: Chiesi
Company Location: Europe
Company Founding Year: 1935
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Austria, Belgium, Canada, Czech Republic, Denmark, Finland, France, Hungary, Italy, Japan, Netherlands, Norway, Slovenia, Spain, United Kingdom, United States
Active Clinical Trial Count: 7
Recent & Upcoming Milestones
- Protalix Biotherapeutics anticipates FDA action on May 9, 2023 regarding BLA for prx-102 treatment for Fabry disease.
- PDUFA date for prx-102 BLA is April 27, 2021. FDA accepted BLA for priority review in Fabry disease treatment.
- FDA accepted BLA for pegunigalsidase alfa for Fabry disease with PDUFA action date set for January 27, 2021. Priority review granted.
Highest Development Phases
Phase 3: Fabry Disease|Proteinuria
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT03614234 |
CLI-06657AA1-03 | P3 |
Completed |
Proteinuria|Fabry Disease |
2026-04-13 |
32% |
2026-05-23 |
|
2022-503128-29-00 |
CLI-06657AA1-01 | P3 |
Recruiting |
Fabry Disease |
2029-12-31 |
2025-05-02 |
Treatments |
|
NCT06328608 |
FLY | P3 |
Recruiting |
Fabry Disease |
2028-10-01 |
34% |
2025-09-20 |
Primary Endpoints |
jRCT2031230079 |
jRCT2031230079 | P3 |
Not yet recruiting |
Fabry Disease |
2027-12-31 |
|||
NCT05710692 |
RISE | P3 |
Recruiting |
Fabry Disease |
2027-10-01 |
33% |
2025-11-26 |
Patient Enrollment|Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
2024-516735-27-00 |
CLI-06657AA1-03 | P3 |
Active, not recruiting |
Fabry Disease |
2025-12-01 |
2025-05-02 |
Treatments |
|
NCT03566017 |
CLI-06657AA1-04 | P3 |
Completed |
Fabry Disease |
2025-01-21 |
33% |
2025-05-21 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |