Luxturna: The Most Expensive One-Time Eye Drug
Luxturna is a gene therapy approved by the U.S. Food and Drug Administration (FDA) in 2017 to treat patients with biallelic RPE65-mediated inherited retinal dystrophy (IRD). This one-time treatment has a list price of $850,000 per patient for both eyes, or $425,000 per eye.
How Luxturna Works
Luxturna addresses the genetic cause of RPE65-mediated IRD, where a faulty RPE65 gene prevents the production of a protein vital for the retina to process light, leading to progressive vision loss. The therapy delivers a functional copy of the RPE65 gene into retinal cells through a subretinal injection, potentially improving functional vision.
Comparison with Other High-Cost Ophthalmic Drugs
While Luxturna is the most expensive one-time eye drug, other ophthalmic medications for conditions like wet age-related macular degeneration (AMD) and diabetic macular edema (DME), also have significant costs, especially over time.
High-Cost Ophthalmic Treatment Comparison
| Feature | Luxturna | Vabysmo (faricimab-svoa) | Eylea (aflibercept) |
|---|---|---|---|
| Cost Structure | ~$850,000 per one-time treatment per patient | ~$2,290 per injection, recurring | ~$1,850 per injection, recurring |
| Indication | Biallelic RPE65-mediated IRD | Wet AMD, DME | Wet AMD, DME |
| Mechanism | Gene therapy delivering functional RPE65 gene | Anti-VEGF and anti-Ang-2 activity | Anti-VEGF activity |
| Frequency | One-time per eye | Every 1-4 months | Every 1-4 months |
| Administration | Subretinal injection in a surgical setting | Intravitreal injection in an office setting | Intravitreal injection in an office setting |
Some treatments for ophthalmic cancers can also exceed $1 million annually.
The Justification for High Gene Therapy Costs
The high cost of Luxturna is often attributed to the small patient population (1,000-2,000 in the U.S.) for this rare condition, meaning development costs are spread over a limited market. The complex research, clinical trials, and manufacturing for gene therapy also contribute to the price. As a one-time treatment aiming for a potentially long-term benefit, it can be seen as a long-term investment compared to ongoing therapies. Manufacturers also offer payment models and patient assistance programs to help manage the cost.
Conclusion: The Impact of High-Cost Eye Drugs
The existence of what is the most expensive eye drug on the market, Luxturna, demonstrates progress in treating rare genetic vision loss with gene therapy. While it offers a potential breakthrough, its high cost brings up issues of healthcare access and affordability. The debate around the cost involves balancing potential significant, lasting benefits with the financial strain on individuals and healthcare systems. Patient assistance and alternative payment models are crucial for making these treatments available.
You can read more about the UPMC Vision Institute's experience with Luxturna here.
