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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