The FDA are currently considering the application for the approval of the first ever gene therapy in the United States, produced by drug developer Spark Therapeutics.

RareDR writes, the FDA have granted rare paediatric disease designation to Luxturna, a gene therapy currently under review for treating RPE65-mediciated inherited retinal dystrophy.

The developers believe that Luxturna has the potential to be the first pharmacologic treatment for biallelic RPE65-mediated inherited retinal dystrophy (IRD). Side effects of this gene mutation include frequently suffering from night blindness (nyctalopia) as a result of decreased light sensitivity during childhood or early adulthood, as well as involuntary back-and-forth eye movements (nystagmus).

In a press release, chief executive officer of Spark Therapeutics, Jeffrey Marrazzo, said, “FDA acceptance for filing of our BLA for Luxturna is an important development for people living with RPE65-mediated IRD, a significant milestone for the gene therapy field, and a strong testament to the dedication of our collaborators and employees.

“As we work closely with FDA in the months ahead, we will remain steadfast in our commitment to bring this important investigational therapy to people living with RPE65-mediated IRD who currently have no pharmacologic treatment options.”

In a Phase 3 trial, the drug has been proven to be safe and effective in improving functional vision in patients with the condition.

Gordon Gund, co-founder and chairman emeritus, Foundation Fighting Blindness (FFB), a non-profit organisation focused on research for preventing and treating blindness caused by IRDS, explains, “Today’s announcement is an important and exciting step forward in the effort to treat blindness caused by inherited retinal diseases. While we await the FDA review process, it is fitting to note that FFB is proud to have been a part of the early support of the research that led to the development of this potential therapy.”