UPDATED: Amgen gets a big win with FDA OK for PCSK9 cholesterol drug Repatha

Amgen ($AMGN) scored the big win for its PCSK9 drug Repatha (evolocumab). The FDA announced Thursday evening that it has given Amgen a green light on marketing the PCSK9 cholesterol drug, opening the door to what will likely prove a blockbuster market.

Amgen's approval came in looking remarkably similar to the rival Praluent's OK, getting approved for patients who don't respond to maximally tolerated statins.

Amgen says it will price its drug at $14,100 a year, or $1.36 a day less than Praluent, giving the company a chance to aim high and negotiate down as needed. Some analysts had initially estimated that the price would hit around $7,500 a year. Sanofi ($SNY) and Regeneron ($REGN) came up with a $40 per day wholesale price ($14,600 a year) for Praluent, which nabbed the first approval for a PCSK9 drug at the FDA.

"With limited differentiation between the Praluent and Repatha labels, and close to parity pricing, we expect both entrants to focus on growing the overall class utilization versus a reliance on short-term contracting wins ahead of cardiovascular outcomes data before YE17," notes Barclays analust Geoff Meacham. "Either way, we continue to expect payors to aggressively manage utilization."

In a long lineup of studies involving thousands of patients, the rivals demonstrated that their drugs have a dramatic impact on bad cholesterol, driving down the numbers in ways that will likely benefit at-risk cardio patients. The drugs are being directed at patients who don't respond to cheap statins, a major consideration for the agency as it faces a growing chorus of critics alarmed at the price new drugs aimed at mass markets are able to fetch these days.

Nomura's analysts recently echoed another theme around today's decision, wondering how the breadth of the label will compare to Praluent's. Skipping the frontline market and moving into second line therapy for statin intolerance (well beyond a restriction to rare cases of familial hypercholesterolemia) was worth a $2.5 billion peak sales estimate--and that includes the hefty discounts that will like play out as the rivals compete for market share in much the same way that companies have been competing in hep C recently.

The theme in the markets now is that Regeneron/Sanofi gave themselves plenty of wiggle room when it comes to discounting their price for key payers. Amgen will now jump into the game as well, and when the dust settles insurers will wrest every advantage they can find.

"Amgen is sensitive to the concerns of payers around cost, budget predictability and paying for value," said Anthony C. Hooper, executive vice president of Global Commercial Operations at Amgen. "We are confident in the ability of Repatha to demonstrate real-world effectiveness and value based on intensive LDL cholesterol reductions, and we will be working with payers and other purchasers to provide innovative pricing programs linking the net price of Repatha to the expected LDL cholesterol reductions and anticipated appropriate patient utilization. By partnering with payers to implement these programs, we can help ensure that all appropriate patients who could benefit from Repatha will have access to this important new therapy."

More help may be on the way for consumers. Pfizer ($PFE) has its own small molecule-blocking PCSK9 drug in the pipeline, and the biotech Esperion believes it can jump into the competition with its LDL-lowering pill, hoping to swoop in and carve a slice out of the market after an upcoming Phase III study.

The horse race to the finish line by the three leading companies has had major implications for each. Amgen has had a few successes, buying Kyprolis and recently expanding the market range for a combo approach against multiple myeloma, for example. There was an approval for the heart drug ivabradine (Corlanor), which many analysts just shrugged off as a marginal new player unlikely to get close to blockbuster status.

Repatha, though, could help add some badly needed luster to the company's R&D group, which has been restructured as Amgen sought to lower the percentage of cash that is committed to drug development.

Regeneron ($REGN), meanwhile, has been adding to its operations in a big way, building on its success with Eylea. And Sanofi, which paid heavily for a seat at Regeneron's table, got a blockbuster contender after a series of internal setbacks and reorganizations of its own.

- here's the release from the FDA

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