Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65

PHILADELPHIA, Nov. 6, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that its lead product candidate, SPK-RPE65, has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA). Breakthrough therapy designation is intended to expedite the development and review of investigational therapies for serious or life-threatening conditions where initial clinical evidence demonstrates a significant improvement over existing therapies. SPK-RPE65 targets a group of blinding conditions known as inherited retinal dystrophies (IRDs) caused by autosomal recessive mutations in the RPE65 gene, and for which there is currently no pharmacologic treatment available. 

"FDA's breakthrough therapy designation for SPK-RPE65 underscores the serious unmet medical needs faced by patients with genetic blinding conditions," said Jeffrey D. Marrazzo, co-founder and chief executive officer, Spark Therapeutics. "We look forward to working closely with FDA to facilitate the review of SPK-RPE65 as we work to bring this potentially transformative treatment to patients."

SPK-RPE65 received breakthrough therapy designation for the treatment of nyctalopia, or night blindness, in patients with Leber's congenital amaurosis due to mutations in the RPE65 gene. The designation was supported by two earlier clinical trials where results to date and reports from the clinical study team suggest that SPK-RPE65 enables patients to perform activities of daily living with greater independence than prior to treatment, and has long-lasting effects in restoring functional vision.  Notably, as reported by the clinical study team, following a single injection of SPK-RPE65 in one eye, children in the trials no longer depended on visual aids to carry out classroom activities and were able to walk and play more like normally sighted children.  

SPK-RPE65 currently is in a fully-enrolled pivotal Phase 3 clinical trial. Spark expects to report data from this Phase 3 trial in the second half of 2015.

The receipt of breakthrough therapy designation for SPK-RPE65 follows several recent and significant corporate developments for Spark as it moves toward completing clinical development of SPK-RPE65:

• The appointment of gene therapy pioneer and former director of The Children's Hospital of Philadelphia's Center for Cellular and Molecular Therapeutics, Katherine High, M.D., as its president and chief scientific officer.
• Key leadership and division head appointments across finance, clinical research and development, technical operations, translational research, human resources and regulatory affairs and quality, including former leaders from Optimer, ViroPharma, Shire, Pfizer, and Eli Lilly.
• The establishment of Spark's new corporate headquarters at 3737 Market Street, Philadelphia, scheduled to be completed in the fourth quarter of 2014.

For more information on the SPK-RPE65 clinical program and the recent expansion of Spark's leadership team, please visit www.sparktx.com.

About Breakthrough Therapy Designation

Breakthrough therapy designation was sanctioned as part of the Food and Drug Administration Safety and Innovation Act in 2012. It is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. For more information please visit: http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/default.htm.

About SPK-RPE65

Spark's most advanced product candidate, SPK-RPE65, is in a fully-enrolled pivotal Phase 3 clinical trial having previously demonstrated the potential to safely restore functional vision in patients with inherited retinal dystrophies (IRDs) caused by mutations in the RPE65 gene. The results of two earlier trials suggest that SPK-RPE65 enables patients to perform activities of daily living with greater independence than prior to treatment and has long-lasting effects, with subjects having been followed for a period of at least five years. Notably, as reported by the clinical study team, following a single injection of SPK-RPE65 in one eye, children no longer depended on visual aids to carry out classroom activities and were able to walk and play more like normally sighted children. Spark expects to report data from its Phase 3 trial in the second half of 2015 and file a Biologics License Application with FDA in 2016. To learn more visit www.sparktx.com/pipeline. 

About Spark Therapeutics     

Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark's initial focus is on treating orphan diseases where no, or only palliative therapies, exist. Spark's most advanced product candidate, SPK-RPE65, is in a fully-enrolled pivotal Phase 3 clinical trial for the treatment of a rare blinding condition. Spark is leveraging SPK-RPE65 to address a broad spectrum of blinding conditions, and also has established a pipeline of gene therapy candidates to treat hematologic and neurodegenerative disorders. Spark's integrated gene therapy platform builds on two decades of research, development and manufacturing at The Children's Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. To learn more visit www.sparktx.com.

SOURCE Spark Therapeutics

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http://www.sparktx.com