Knight Therapeutics intends to find out how much drugmakers are willing to pay for the FDA's attention, putting its priority review voucher up for sale and shining a light on a program designed to encourage neglected disease R&D.
Inspired by BioMarin ($BMRN), which made $67.5 million by flipping its coupon for a speedy agency review to Sanofi ($SNY) and Regeneron ($REGN) last month, Knight is auctioning off a voucher it received for successfully developing a treatment for the tropical disease leishmaniasis earlier this year.
Launched in 2007, the FDA's voucher system is meant to spur work in tropical and rare pediatric diseases, offering a fast-track pass to any company that wins approval for a qualifying therapy. When redeemed, the voucher shortens the review process from 10 to 6 months, granting the holder special access to senior agency officials.
Most important, the credit can be bought, sold or traded, and the program's inventors theorized it could be worth hundreds of millions of dollars to the right buyer.
That, of course, hasn't panned out. BioMarin's credit, won after the FDA approved Vimizim for the rare Morquio A syndrome in February, failed to crack the $100 million threshold, and it's hard to imagine a more seller-friendly scenario: Sanofi and Regeneron were trailing rival Amgen ($AMGN) in a blockbuster race to commercialize new cholesterol drugs, and a quicker approval would give the duo first crack at what's expected to be a multibillion-dollar market.
Furthermore, a faster review is no guarantee of success, as the first voucher winner found out. In 2011, Novartis ($NVS) chose to hold onto the coupon it won for developing the malaria treatment Coartem, applying it to a supplemental filing designed to expand the label of the arthritis treatment Ilaris. The FDA held up its end of the bargain and, within 6 months, rejected the application.
But despite all that, Knight believes it can sway a buyer to pay for its voucher and thereby "set the bar high to encourage others to invest in R&D for neglected tropical diseases," CEO Jonathan Ross Goodman said.
"We now have one data point on the voucher's value and remain the only company with a voucher who has publicly stated an intention to sell it," Goodman said in a statement. "A Big Pharma may want our voucher if they are behind in a race to market and want to catch up, or if they are ahead in a race and don't want their competitor to hop on a faster bus."
If that doesn't work out, it could be because the FDA's recent efforts to be flexible with drug developers have cannibalized the vouchers' value. Over the past few years, the agency has unveiled standalone fast-track, breakthrough-therapy and accelerated-approval designations, which it confers to drugs that treat serious unmet needs. At the same time, a treatment's market value tends to dovetail with those same criteria, meaning the drugmakers that stand to benefit most from buying a voucher may very well already have one of the agency's other, equivalent guarantees.
Knight is one of just four companies to receive a voucher, a group that includes BioMarin, Novartis and Johnson & Johnson ($JNJ), which picked one up in 2012 when the FDA approved Sirturo (bedaquiline) for tuberculosis. The agency isn't required to disclose which other companies have requested vouchers, a spokeswoman said.
- read Knight's statement