Gene expression
What’s new about pegunigalsidase alfa for Fabry?
Open access · cc by · source: Europe PMC
This review covers plant-cell PEGylated α-Gal A ERT: Gb3 clearance, phase 3 programs, and BALANCE eGFR slope difference −0.36 mL/min/1.73 m²/year vs agalsidase beta within non-inferiority bounds.
Study at a glance
- Design
- Other — Mini-review of pegunigalsidase alfa pharmacology and phase 1–3 Fabry ERT trials
- N
- N=3 · Focus on three phase 3 programs: BRIDGE, BRIGHT, BALANCE
- Population
- Patients with Fabry disease in reviewed clinical trials of pegunigalsidase alfa
- Outcome
- Renal function (eGFR slope), Gb3 clearance, safety/tolerability vs prior ERTs
Structured fields used in claim comparison tables when every cited study has a complete layer.
Key findings
Enhanced stability/half-life via PEGylation; favorable efficacy/safety signals with caveats for uncontrolled BRIDGE/BRIGHT; BALANCE CI above −3 and including zero; 97% BALANCE completers continued therapy.
Methodology
Summarized pharmacology and phase 1/2 plus BRIDGE/BRIGHT/BALANCE evidence, including BALANCE ITT eGFR slopes and extension uptake.
Limitations
Review does not replace reading primary trial protocols; BRIDGE/BRIGHT lack controls.
How this study connects
Role on claims
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Not yet placed on a claim. This paper has study layers, but no concept page yet cites it as support, challenge, or qualifier.
Related papers in this topic
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