Expanding Access to Sickle Cell Gene Therapy

Briefing Document

📋 Page Properties

🔖 Table of Contents

1. Executive Summary
2. Key Themes
   1. Transformative Potential
   2. Barriers to Access
   3. CMS CGT Access Model
3. Conclusion

Executive Summary

Why this matters: Two FDA‑approved gene therapies—exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia)—offer a potential cure for sickle cell disease (SCD). Yet list prices of $2.2–3.1 million per treatment threaten to widen health‑equity gaps.

CMS’s new Cell & Gene Therapy (CGT) Access Model seeks to close those gaps by striking outcomes‑based agreements (OBAs) with manufacturers, delivering guaranteed discounts and wrap‑around patient supports across 33 states, D.C., and Puerto Rico.

Key Themes

A. Transformative Potential of Gene Therapy

• Curative intent: Unlike hydroxyurea or voxelotor, these autologous stem‑cell therapies target the genetic root of SCD.
• Mechanisms
  • Casgevy: CRISPR/Cas9 editing of BCL11A erythroid enhancer → ↑ fetal hemoglobin.
  • Lyfgenia: Lentiviral gene addition of HbAT87Q → produces anti‑sickling β‑globin.
• Regulatory milestone: Simultaneous FDA approvals (Dec 8 & Dec 15 2023).
• Clinical results: Lyfgenia produced complete resolution of vaso‑occlusive crises in 88 % of phase 3 participants within 6–18 months.