Spark Therapeutics, the developer of the drug, believes that Luxturna has the potential to be the first pharmacologic treatment for biallelic RPE65-mediated inherited retinal dystrophy. The drug was granted Priority Review and has a Prescription Drug User Fee Act (PDUFA) date of January 12, 2018.
Previously untreatable, RPE65-mediated inherited retinal dystrophy is an inherited retinal disease (IRD), and a natural history study has shown that people with it eventually progress to total blindness.
People living with IRDs due to biallelic RPE65 gene mutations frequently suffer 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).
“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,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics.
“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.”
Luxturna is Spark’s most advanced investigational candidate and is currently under Priority Review with FDA for the treatment of RPE65-mediated IRD. The potential treatment option has also previously received breakthrough therapy and orphan product designations from the FDA and orphan product designations from the European Medicines Agency (EMA).