The ophthalmology market is on the verge of a gene therapy breakthrough, with the potential for transformative therapies to generate blockbuster revenues in the near future.

On July 17, 2017 Spark Therapeutics announced that the FDA had granted priority review for voretigene neparvovec a one-time therapy, for patients with vision loss due to RPE65-mediated inherited retinal disease.

It also announced the proposed brand name of Luxturna.

In the first ever successful randomized, controlled Phase III trial to be completed for a gene therapy for a genetic disease, Luxturna was trialed in 31 patients, and was found to significantly improve functional vision and light sensitivity in subjects, with no serious side effects.

If approved, the drug will become the first approved gene therapy in the US, and would be the only pharmacological treatment available for RPE65-mediated inherited retinal disease.

Luxturna set for strong commercial success

GBI Research expects Luxturna to be approved in 2017, and to generate $33m in its first year on the market.

Across the forecast period revenue from Luxturna is forecast to increase at a CAGR of 64.6%, reaching $646m in 2023.

If Luxturna achieves GBI’s forecast it will be the eight most successful product in ophthalmology in 2023, despite being a once-only orphan drug.

Gene therapies in other indications

GBI Research has identified 79 gene therapies in active development across 17 ophthalmology indications – making it the second most common drug type in ophthalmology.

The majority of products are at the Preclinical or Discovery stages, and Luxturna is the only gene therapy in ophthalmology to have reached the Pre-registration stage.

The most common ophthalmology indications for which gene therapies are being developed are Retinitis pigmentosa and wet age related macular degeneration (wAMD).

The prevalence of wAMD is particularly high, so if a gene therapy achieves approval for this disease it will undoubtedly go on to achieve blockbuster status.