BioMarin Sells Priority Review Voucher To Regeneron


By Ron Winslow

Regeneron Pharmaceuticals Inc. ( REGN ) and Sanofi SA (SAN.FR) are spending $67.5 million on a novel bet they hope will help them speed their experimental cholesterol drug to the market.

The companies are paying the money to acquire a special voucher held by BioMarin Pharmaceuticals Inc. ( BMRN ) in a bid to hasten regulatory review of their drug alirocumab, one of an emerging class of medicines that lower cholesterol by targeting a gene known as PCSK9.

BioMarin was awarded the voucher early this year as part of an incentive program established by the U.S. Food and Drug Administration to encourage development of drugs for rare pediatric diseases. The voucher entitles the holder to ask the FDA for priority review of a drug application that would otherwise get a standard review. That could shorten the review process to six months from the standard 10 months.

BioMarin received the voucher in conjunction with FDA approval of Vimizim, a treatment for a rare pediatric condition called Morquio A syndrome that afflicts about 800 patients in the U.S. While BioMarin could have used the voucher itself, the program also allows companies to sell a voucher to another company. The voucher does not need to be used on a drug for a rare pediatric condition.

The voucher was the first to be issued under the pediatric incentive program, and also the first to change hands. A separate program to encourage development of medicines for rare tropical diseases already existed.

Regeneron, along with Sanofi, and BioMarin separately announced the deal for the voucher on a day when Regeneron and Sanofi also disclosed that alirocumb successfully achieved significant cholesterol reduction in nine late-stage trials.

Whether the voucher will lead to faster approval of the drug isn't certain.

"It's going to be tough to speculate," Christopher Viehbacher, chief executive of Sanofi, said in an interview. But the analysis of data from the nine studies "gives us a lot of confidence in the approvability of the drug."

The companies released few details of the data, which are expected to be released in full at upcoming medical meetings, including the European Society of Cardiology congress in Barcelona at the end of August.

--Joseph Walker contributed to this article.

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