Amgen ($AMGN) just lost a key regulatory race. Armed with a handy FDA shortcut voucher, Regeneron ($REGN) and its Big Pharma partner Sanofi ($SNY) announced today that the agency has accepted their application for the powerful new PCSK9 cholesterol drug alirocumab and set a priority deadline of July 24 for their marketing decision. If they get a quick green light, as many analysts expect, their decision to buy the priority review voucher will be rewarded with first-mover advantage with U.S. payers as Amgen sits out an FDA game clock that ends August 27.
There are still plenty of 'ifs' to acknowledge. An approval is not assured, the FDA doesn't always abide by its deadlines and a few weeks head start won't necessarily translate into big market gains anyway. But Sanofi and Amgen both see these cholesterol drugs as major new product opportunity and proof positive that their pipeline strategies can deliver. Regeneron wants to demonstrate that it can build a portfolio of big products and both development teams recruited thousands of patients to provide a mountain of safety and efficacy data for the regulators.
Sanofi and Regeneron plan to market their new drug as Praluent.
The PCSK9 showdown between these two competing programs also dramatically underscores a new reality for drug developers. Gilead's ($GILD) decision to charge a steep price for its revolutionary new hep C drugs gave AbbVie ($ABBV) a chance to snag an exclusive place on some big formularies at payers, which direct prescribing rules for tens of millions of Americans. According to Express Scripts, which has led the charge for deep discounts, this new cholesterol drug class has already triggered some negotiations over pricing.
|Express Scripts Chief Medical Officer Steve Miller|
This new drug class "is the next opportunity for us," Express Scripts Chief Medical Officer Steve Miller told Bloomberg recently. And contrasting these companies' marketing strategies with Gilead, he added that "they are working with us."
Sanofi and Regeneron got their priority voucher from BioMarin for $67.5 million, which had in turn won it under a new federal program aimed at spurring the development of drugs for rare diseases.
These three companies aren't the only PCSK9 players in the pipeline. Pfizer ($PFE) has a big Phase III program of its own that often goes overlooked when analysts size up the horse race between the leaders. The pharma giant also highlighted at the JP Morgan conference that it's working on a preclinical oral drug that could prove much easier to take than the injectables now rushing toward the drug market. And other early-stage PCSK9 programs have been launched by companies like Alnylam.
One of the reasons why there's so much competition here at the R&D finish line is that investigators believe that the science behind these therapies is well understood. But once the mists of uncertainty are cleared away in drug development, the need to advance new products after a whole generation of blockbusters has gone generic also spurs plenty of competition for new markets, a trend that will inevitably blunt the ambitious sales forecasts that are being laid out for some of these would-be blockbusters.
- here's the release