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PRX-102 (pegunigalsidase alfa)
PRX-102 is a pegylated recombinant human α-galactosidase A enzyme that breaks down accumulated globotriaosylceramide (Gb3) in cells affected by Fabry disease.
PRX-102 is a pegylated recombinant human α-galactosidase A enzyme that breaks down accumulated globotriaosylceramide (Gb3) in cells affected by Fabry disease. Used for Fabry disease (in development).
At a glance
| Generic name | PRX-102 (pegunigalsidase alfa) |
|---|---|
| Also known as | pegunigalsidase alfa, Recombinant human alpha galactosidase-A |
| Sponsor | Protalix |
| Drug class | Enzyme replacement therapy (ERT) |
| Target | α-galactosidase A (GLA) |
| Modality | Biologic |
| Therapeutic area | Rare genetic disease / Lysosomal storage disorder |
| Phase | Phase 3 |
Mechanism of action
PRX-102 is an enzyme replacement therapy (ERT) designed to address the deficiency of α-galactosidase A in Fabry disease patients. The pegylation extends the drug's half-life and reduces immunogenicity compared to non-pegylated versions. By replacing the missing or deficient enzyme, PRX-102 catalyzes the hydrolysis of Gb3 and related glycosphingolipids, reducing their pathological accumulation in tissues and potentially slowing disease progression.
Approved indications
- Fabry disease (in development)
Common side effects
- Infusion-related reactions
- Headache
- Pyrexia
- Nausea
Key clinical trials
- Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease (PHASE3)
- A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (PHASE2, PHASE3)
- Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (PHASE2, PHASE3)
- Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (PHASE3)
- Extension Study of PRX-102 for up to 60 Months (PHASE1, PHASE2)
- Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function (PHASE3)
- Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) (PHASE3)
- Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- PRX-102 (pegunigalsidase alfa) CI brief — competitive landscape report
- PRX-102 (pegunigalsidase alfa) updates RSS · CI watch RSS
- Protalix portfolio CI