General Science Archives | 17 July 2017

Industry News: Spark Therapeutics’ Biologics License Application for Investigational Voretigene Neparvovec Accepted for Filing by FDA

A massive step forward for the Retina Community as The FDA grants  a Priority Review with Prescription Drug User Fee Act (PDUFA) date of Jan. 12, 2018

LUXTURNATM (voretigene neparvovec) unveiled as proposed trade name.

PHILADELPHIA, July 17, 2017 Spark Therapeutics. a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the Biologics License Application (BLA) and granted Priority Review for voretigene neparvovec, an investigational, potential one-time gene therapy candidate for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). The investigational gene therapy, which has the proposed trade name LUXTURNA™ (voretigene neparvovec), has the potential to be both the first pharmacologic treatment for IRD and the first gene therapy for a genetic disease in the United States.

Priority Review is granted to therapeutics that would offer major advances over existing therapies or would provide a treatment where no adequate therapy exists. FDA’s goal for taking an action on Priority Review applications is six months. For the review of LUXTURNA, FDA has assigned a PDUFA date of 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,” 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.”

Investigational LUXTURNA is intended to be administered one time per eye in patients with vision loss due to biallelic RPE65-mediated IRD. A natural history study has shown that people with this IRD eventually progress to complete blindness.

“Today’s announcement is an important and exciting step forward in the effort to treat blindness caused by inherited retinal diseases,” commented Gordon Gund, co-founder and chairman emeritus, Foundation Fighting Blindness (FFB), a nonprofit organization focused on research for preventing and treating blindness caused by IRDs. “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.”

Christina Fasser, President of Retina International said of the announcement “Today we have reached a milestone in the development of cures and treatments for Inherited Retinal Degenerations. This news represents a significant step forward for our community. Retina International will follow the review process with interest and we look forward to updating our members on progress”.


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