Spark Therapeutics has sold its Rare Pediatric Disease Priority Review Voucher (PRV) to Jazz Pharmaceuticals for US$110 Million (€92 million).
Spark Therapeutics received the PRV from the US Food and Drug Administration (FDA) when its gene therapy for inherited retinal disease, Luxturna (voretigene neparvovec), was approved.
But the firm disclosed in an SEC filing it is selling the voucher to Ireland-headquartered biopharma firm Jazz to help support its future R&D.
“We will invest the proceeds from the sale back into the research and development of our robust pipeline of investigational gene therapies that may provide benefits for people with limited treatment options,” Spark spokesperson Monique Da Silva told BioProcess Insider.
The voucher will allow Jazz to accelerate the approval process for one of its drug candidates, with the FDA reviewing the product in six months rather than the standard 10 months.
“The PRV could benefit patients as we look to bring new medicines to market where additional treatment options are needed and sometimes no medicines are available,” Jazz spokesperson Andrew Civers-Davis told us.
“We have not decided on our plans for the voucher, but we could use the PRV for a product candidate in our current pipeline, or it could be used for a future asset from our corporate development efforts.”
The FDA approved Luxturna in December 2017 making it the first gene therapy product to receive regulatory the thumbs up for a genetic disease.
The therapy is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells.
“The AAV vector is used to transport the functional RPE65 gene, which is injected into the retina,” Da Silva said.
Luxturna is manufactured at Spark’s manufacturing facility in West Philadelphia, which Da Silva said is the first licensed manufacturing facility in the US for a gene therapy treating an inherited disease.