FDA-Approved Gene Therapies
| Therapy | Indication | Approved | List Price |
|---|---|---|---|
| Hemgenix (etranacogene dezaparvovec) | Hemophilia B | 2022 | $3.5M |
| Zynteglo (betibeglogene autotemcel) | Beta-thalassemia | 2022 | $2.8M |
| Skysona (elivaldogene autotemcel) | Cerebral ALD | 2022 | $3.0M |
| Casgevy (exagamglogene autotemcel) | Sickle cell disease | 2023 | $2.2M |
| Lyfgenia (lovotibeglogene autotemcel) | Sickle cell disease | 2023 | $3.1M |
The Pipeline
The gene therapy pipeline includes hundreds of therapies in clinical trials for conditions including Duchenne muscular dystrophy, Huntington's disease, Parkinson's disease, and various cancers. The FDA expects to approve 10–20 new gene therapies per year by 2030. Employer health plans must prepare for the financial impact of these approvals.
The One-Time Treatment Promise
IQ Insight
Gene therapies are typically one-time treatments with the potential for lifelong benefit. A hemophilia patient who receives Hemgenix may never need factor replacement therapy again — saving $500,000–$1 million per year in ongoing treatment costs. The $3.5 million price tag may be justified by the lifetime savings — but the upfront cost is a significant challenge for employer health plans.