The Approval of Luxturna: The First Gene Therapy for Inherited Retinal Disease

Luxturna, the first gene therapy approved for the treatment of inherited retinal disease, is a significant breakthrough in the field of gene therapy. In December 2017, the FDA approved Luxturna for the treatment of patients with biallelic RPE65 mutation-associated retinal dystrophy.

Here's what you need to know about Luxturna:

1. How Luxturna works: Luxturna is a one-time gene therapy that delivers a functional copy of the RPE65 gene to retinal cells using a viral vector. The functional copy of the gene then helps the cells produce the necessary protein to convert light into an electrical signal that the brain can interpret.
2. Clinical trials: Luxturna's approval was based on data from clinical trials that showed significant improvements in vision for patients with biallelic RPE65 mutation-associated retinal dystrophy. Patients who received Luxturna showed improvements in both light sensitivity and visual acuity compared to those who did not receive the therapy.
3. Administration: Luxturna is administered through subretinal injection in a single eye surgery.
4. Safety and side effects: The most common side effects of Luxturna are eye redness, cataracts, and increased intraocular pressure. However, these side effects are usually mild and transient.
5. Cost: Luxturna is one of the most expensive therapies in the world, with a price tag of $850,000 per eye. The high cost of the therapy has raised concerns about access and affordability.

Luxturna's approval marks a significant milestone in the development of gene therapies for inherited diseases. The therapy offers hope to patients with biallelic RPE65 mutation-associated retinal dystrophy and their families, who previously had no treatment options. While the high cost of the therapy is a concern, it is hoped that as more gene therapies are developed, costs will come down, making these life-changing treatments more accessible to those who need them.

In conclusion, Luxturna's approval is a significant step forward for the field of gene therapy and a promising development for patients with inherited retinal diseases. As more gene therapies are developed, it is hoped that they will be able to provide similar life-changing treatments for other genetic diseases.

‍