Rare Daily Staff

Spark Therapeutics said it has entered into an agreement to sell its rare pediatric disease priority review voucher to Jazz Pharmaceuticals for $110 million.

The U.S. Food and Drug Administration awarded Spark with the pediatric review voucher with the approval of its gene therapy Luxturna as a treatment for confirmed biallelic RPE65 mutation-associated retinal dystrophy, an inherited form of vision loss that may result in blindness. Luxturna was the first gene therapy approved in the United States that targets a genetic disease.

FDA grants Rare Pediatric Disease Priority Review Vouchers as a way to encourage the development of treatments for rare pediatric diseases. The vouchers are transferable and can be used to accelerate the review of large market therapeutics. The priority review voucher entitles the holder to priority review of a single New Drug Application or Biologics License Application.

“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 La Barge, chief legal officer, Spark Therapeutics.

The Spark PRV sale represents a downtick in recent similar transactions. In November, BioMarin sold a PRV for $125 million and in December, Ultragenyx sold one for $130 million.
Spark said the transaction is subject to customary closing conditions.

April 30, 2018
Photo: Joseph La Barge, chief legal officer, Spark Therapeutics

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