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Member News from Spark Therapeutics

Voucher sale brings $110M payday for Spark Therapeutics
By  – Senior Reporter, Philadelphia Business Journal

Spark Therapeutics is taking advantage of a perk in a Food and Drug Administration program for a nine-figure payday.

The Philadelphia gene therapy company said Monday it has sold its rare pediatric disease priority review voucher to Jazz Pharmaceuticals for $110 million. Spark will receive the payment once the transaction closes.

Spark received the voucher after the FDA approved the company’s new drug application for Luxturna in December. Luxturna is its gene therapy treatment for an inherited retinal disease that leads to blindness if untreated.

Companies become eligible for the voucher when they receive a rare pediatric disease designation for a new drug candidate. The designation is given to experimental therapies targeting serious or life-threatening diseases that affect fewer than 200,000 individuals in the United States.

The voucher can be used by a company to receive an accelerated review of any subsequent new drug application. It can be worth big money for a company that is in a race with competitors to develop a new drug candidate for a particular condition. The FDA allows such vouchers to be sold as an inducement to get biopharmaceutical companies to pursue new therapies for rare pediatric conditions.

“The sale of our [priority review voucher] 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 of Spark Therapeutics (NASDAQ: ONCE), in a statement.

Jazz Pharmaceuticals is based in Dublin, Ireland, and has U.S. operations in Philadelphia; Ewing, N.J., and Palo Alto, Calif. The company focuses on developing products for cancer and blood disease and sleep disorders.

The Regulatory Affairs Professionals Society (RAPS), a global organization whose members include the makers of medical devices, medicines and nutritional products, tracks the sale of vouchers. According to RAPS, 18 vouchers have been issued since 2009 and six have been sold. Abbvie Inc. of Chicago paid $350 million for a voucher in 2015, the highest price to date. Others have sold to companies including Sanofi and Gilead Sciences for between $67 million and $245 million.

Fibrocell (NASDAQ: FCSC), an Exton cell and gene therapy company, has received two rare pediatric disease designations – giving it the potential to be awarded two vouchers if both products receive FDA approval.

In an interview last year, Fibrocell CEO John Maslowskisaid vouchers attract a lot of interest when put on the auction block.

“A voucher can accelerate a review from 10 months to six months,” Maslowski said. “For a company like us, the great thing is you can sell the voucher to another entity and use proceeds for product development costs. … It’s like a Monopoly card. It’s a nice thing to be able to hold onto.”

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