A $110M jackpot will fund hemophilia R&D at Spark Therapeutics in West Philly

Spark Therapeutics, the Philadelphia-based biotech company, announced Monday that it will receive a $110 million jackpot it will use to fund R&D into new experimental drugs in the pipeline for the bleeding disorder hemophilia.

Spark's windfall comes with its agreement to sell a "priority review voucher" to Jazz Pharmaceuticals. The FDA awarded the voucher to Spark in December after approving the company's gene therapy product Luxturna, which with one injection to an eye treats a rare inherited form of blindness in babies. The cure, which represents the first FDA-approved gene therapy treatment for an inherited condition, runs about $850,000 a child.

The $110 million bonus will be well-spent, a spokeswoman said.

"Spark currently has three other investigational gene therapies in the clinic. We have great confidence in SPK-8011 for hemophilia A and are encouraged by our clinical trial results to date, " said Spark spokeswoman Monique Da Silva. "The FDA recently granted breakthrough therapy designation to SPK-8011," which will speed up FDA reviews.

The treatment has also been granted orphan-disease designation, which includes various development incentives, including tax credits.

The FDA uses priority review vouchers to encourage drug companies to develop medicines to treat rare illnesses in children and tropical diseases. A federal review of a drug can take more than 10 months. A voucher slashes through the red tape, fast-tracking the process. A voucher can shave four months off the time it takes to get the drug approval.

Fewer than two dozen of the vouchers have been awarded since the FDA launched the program in 2009. Companies can use the vouchers themselves or sell them to the highest bidder. The vouchers never expire.  In 2015, Abbvie Inc. paid $350 million to United Therapeutics for a voucher.

Da Silva said the company was not disappointed that the voucher sold for $110 million. "When you look at the more recent sales of [vouchers], you'll note that we are within the current range of sales prices," she said.

A spokesman for Jazz Pharmaceuticals, an Irish biopharmaceutical company with offices at 18th and Market Streets,  said it had not decided on plans for the voucher.

Spark Therapeutics was founded in 2013 by two researchers at the Children's Hospital of Philadelphia. CHOP remains a minority shareholder in the company. Spark is also working on investigational gene therapies for Huntington's disease and Batten's disease, a fatal nervous system condition. Spark logged just $12.1 million in revenue in 2017. Its stock (ONCE) closed at  $76.32 Monday in Nasdaq trading. The company's market cap is about $2.84 billion.

Luxturna is used to treat Leber's congenital amaurosis, an eye disease that effects the retinas of about 1,000 children in the U.S. With a single injection to the retina — at $425,000 a shot — Luxturna restores sight in infants with the hereditary disease. In January, Spark struck a $105 million deal with Swiss drug maker Novartis to sell the medicine outside the country.