Spark Therapeutics Sells Priority Review Voucher for $110 Million

PHILADELPHIA, April 30, 2018 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced it has entered into an agreement to sell its rare pediatric disease priority review voucher (PRV). The PRV was received when LUXTURNA^TM (voretigene neparvovec-rzyl) was approved by the U.S. Food and Drug Administration (FDA).

Under the PRV program, a sponsor who receives FDA approval for a rare pediatric disease drug or biologic may qualify for a voucher to be redeemed at a future time for priority review of a subsequent marketing application for a different product.

“The sale of our PRV will provide an influx of capital to reinvest back into the research and development of our robust pipeline of investigational gene therapies that may provide benefits for people with limited treatment options,” said Joseph W. La Barge, chief legal officer, Spark Therapeutics.

Per the terms of the agreement with Jazz Pharmaceuticals, Spark Therapeutics will receive $110 million upon closing of the transaction, which is subject to customary closing conditions.

About Spark Therapeutics
At Spark Therapeutics, a fully integrated company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia and neurodegenerative diseases. We have successfully applied our technology in the first FDA-approved gene therapy in the U.S. for a genetic disease, and currently have three programs in clinical trials, including product candidates that have shown promising early results in patients with hemophilia. At Spark, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.

Cautionary note on forward-looking statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘predict,’’ ‘‘will,’’ ‘‘would,’’ ‘‘could,’’ ‘‘should,’’ ‘‘continue’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that (i) we may not close the transaction for the sale of our PRV voucher and would not have additional funds to reinvest into our research and development programs.  For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and other filings we make with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Spark Therapeutics undertakes no duty to update this information unless required by law.

Investor Contact:            
Ryan Asay                          
[email protected]
(215) 239-6424

Media Contact:
Monique da Silva
[email protected] 
(215) 282-7470