This article is more than 5 years old. Information may no longer be current.

FDA accepts biologics license application for voretigene neparvovec

Add topic to email alerts

Receive an email when new articles are posted on

Please provide your email address to receive an email when new articles are posted on .

Subscribe

Added to email alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

Back to Healio

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Back to Healio

The FDA has accepted a biologics license application and granted priority review for voretigene neparvovec, according to a press release from Spark Therapeutics.

An investigational gene therapy candidate for vision loss due to biallelic RPE65-mediated inherited retinal disease (IRD), voretigene neparvovec, which has the proposed trade name of Luxturna, could be both the first gene therapy for a genetic disease in the United States and the first pharmacologic treatment for IRD, the release said.

A PDUFA date for the treatment, which is intended to be administered one time per eye, has been set for Jan. 12, 2018.

“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,” Jeffrey D. Marrazzo, CEO of Spark Therapeutics, said in the release. “As we work closely with the 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.”