AstraZeneca nabs priority review voucher as Wellstat bags FDA OK

AstraZeneca's headquarters in London--Courtesy of AstraZeneca

Late last Friday the FDA approved a new drug for an ultrarare condition that is linked to anemia and developmental retardation in children, handing over a priority review voucher to the developer for the pediatric OK which is being passed on immediately to AstraZeneca ($AZN). 

The FDA approved Xuriden--the oral uridine triacetate--for a condition known as hereditary orotic aciduria, which is triggered by a breakdown of a key enzyme leading to an accumulation of orotic acid in the urine. According to the FDA, there are 20 known cases of this condition and the drug--a pill used to replace the lost uridine--was tested in four of them in a singe-arm study.

Not much is known about Gaithersburg, MD-based Wellstat, run by Samuel Wohlstadter. The company stopped updating the news section on its website four years ago but put out a press release Tuesday morning noting that they had already agreed to hand over the priority review voucher to AstraZeneca last fall in a deal with undisclosed terms.

A spokesperson for Wellstat told FierceBiotech that the company plans to start selling the therapy itself once it becomes commercially available next year. And the biotech plans to wait until then to announce the price.

Priority review vouchers, a bonus from the FDA that is intended to help encourage the development of new drugs for pediatric and rare conditions, have been an increasingly pricey commodity on the open market. 

In August, United Therapeutics ($UTHR) sold its priority review voucher to AbbVie ($ABBV) for a record $350 million, just a month after Retrophin ($RTRX) snagged a $245 million voucher deal with Sanofi ($SNY). These vouchers can be used to shave four months off the review process, an advantage that Regeneron ($REGN) and Sanofi used to steal a march on Amgen ($AMGN) in their race to the first U.S. approval of a PCSK9 therapy.

"The FDA's approval of Xuriden for hereditary orotic aciduria is an important breakthrough for patients with this rare, potentially life-threatening orphan disease and for their families," says Wellstat R&D director Michael Bamat in a statement. "There have been no approved treatments for these patients until now."

- here's the FDA release

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