By Fineline Cube 
Chiesi Group announced that the Committee for Medicinal Products for Human Use (CHMP) has recommended approval for pegunigalsidase alfa using a 2 mg/kg every‑four‑weeks (E4W) dosing regimen for adult Fabry disease patients stable on enzyme replacement therapy (ERT). The extended interval builds on the biweekly regimen approved by the European Commission in May 2023.
Regulatory Milestone & Partnership Terms
| Item | Detail |
|---|---|
| Company | Chiesi Group / Chiesi Global Rare Diseases |
| Co‑Developer | Protalix BioTherapeutics (NYSE: PLX) |
| Drug | Pegunigalsidase alfa (pegylated ERT) |
| Indication | Fabry disease |
| CHMP Recommendation | E4W dosing regimen (2 mg/kg) |
| Previous Approval | Biweekly regimen (May 2023) |
| Regulatory Milestone | Protalix eligible for USD 25 million payment upon EC approval |
| Strategic Value | First ERT with flexible dosing options for Fabry patients |
Drug Profile & Clinical Innovation
- Mechanism: Pegylated enzyme replacement therapy delivering α‑galactosidase A to clear globotriaosylceramide (GL‑3) accumulation
- Dosing Advantage: E4W regimen reduces injection burden from 26 to 13 infusions annually, improving patient compliance and quality of life
- Patient Population: Targets adult Fabry patients stable on ERT, representing ~60% of diagnosed population in EU
- Competitive Landscape: Competes with Sanofi’s Fabrazyme (biweekly) and Takeda’s Replagal (EOW); pegunigalsidase alfa’s E4W option offers differentiation
Market Impact & Commercial Outlook
- Fabry Disease Market: EU market valued at $800 million : in 2025, growing at 6% CAGR ; limited treatment options create premium pricing opportunity
- Milestone Impact: $25 million payment to Protalix will support its protein expression platform development and pipeline advancement
- Pricing Strategy: E4W convenience may support 15–20% premium over standard biweekly ERTs
- Strategic Validation: CHMP backing strengthens Chiesi’s rare disease franchise and validates Protalix’s plant‑cell expression technology
- Next Steps: European Commission decision expected Q2 2026; US FDA filing for E4W regimen under discussion
Forward‑Looking Statements
This brief contains forward‑looking statements regarding regulatory approval timelines, milestone payments, and commercial prospects for pegunigalsidase alfa. Actual results may differ due to EC review outcomes, competitive dynamics, and market access negotiations.-Fineline Info & Tech