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A Tale of Two Drugs … One with a Golden (aka Pediatric PRV) Ticket

September 9, 2015 | Mark Cierpial, PhD, RAC, Chief Executive Officer | Regulatory Affairs

In a recent post, I provided some background information on FDA Priority Review Vouchers (PRVs) and the steadily increasing sales prices we’ve been seeing for them over the past year. That post ended with a question … How much is one of these so-called “golden tickets,” good for one priority review by the FDA, really worth?

money Taking a new drug or biologic through the development process can take over a decade and cost hundreds of millions of dollars, so being able to start selling your product as soon as possible is obviously desirable.

That being said, would you pay $350 million for the opportunity to start selling your drug 4 months earlier? You’re probably thinking to yourself, “well, it depends.”
Fair enough … So what if you’re in a horse race with another pharmaceutical company to be first-to-market with a new class of biologic agents to treat a major cardiovascular disease? And what if that new class of drugs was projected to reach peak sales in excess of $20 billion? How much would you pay for a PRV that would cut your BLA review time from 10 months to 6 months? Horses

A New Class of Cholesterol Lowering Drugs

Statin drugs, such as Lipitor®, Crestor®, Zocor®, have long been the go-to treatment for lowering LDL (“bad”) cholesterol, and many studies have demonstrated their beneficial effects on heart attack and stroke prevention, as well as other cardiovascular complications.

Not all patients, however, are able to get their LDL levels under sufficient control with statins, and some must discontinue statin treatment due to intolerable muscle pain.

Stop PCSK9 inhibitors are a new class of biologic drugs that dramatically lower cholesterol levels via a mechanism different from the statins. PCSK9 inhibitors are monoclonal antibodies (MABs) that target a protein, PCSK9, that researchers discovered regulates LDL levels in the blood. PCSK9 actually works against the receptors which transport LDL from the blood into the liver where it is metabolized. Thus, inhibiting PCSK9 leads to more LDL being eliminated from the blood.
Because they work differently than statins, PCSK9 inhibitors can reduce LDL by 50% or more, even if the starting point is at a level already reduced by statins. As such, this new class of cholesterol-lowering drugs is expected to change the landscape for heart attack prevention in the coming years. MoneySymbol GlobalData, a medical analytics company, has estimated that as a class, PCSK9 inhibitors could generate yearly sales of nearly $18 billion by 2023.

The Horse Race

Repatha The first two PCSK9 inhibitors to make it through the development process in the US were Amgen’s Repatha™ (evolocumab) and Sanofi-Aventis’ Praluent® (alirocumab). Praluent

Amgen was the first to submit their BLA, and it was filed by the FDA in late October 2014. That started a 10-month review clock, with a PDUFA action date of August 27, 2015. Sanofi-Aventis submitted their BLA about 3 months after Amgen, and it was filed by the FDA in late January 2015. The PDUFA action date for Praluent should have been November 24, 2015, about 3 months after Amgen’s date for Repatha.

However, recall from the previous post that Sanofi-Aventis purchased a Pediatric PRV from Biomarin in July of 2014. By using the purchased voucher for its Praluent BLA, Sanofi-Aventis’ PDUFA action date became July 24, 2015.

At a cost of $67.5 million, Sanofi-Aventis went from being 3 months behind going into the final turn, to 1 month ahead as they crossed the finish line!


Worth it?

So that brings us back to the original question, what is a PRV really worth?

In the example cited above, Sanofi-Aventis was able to beat Amgen to a multi-billion dollar market for a mere $67.5 million. Conventional wisdom states that being first-to-market is highly desirable, and should translate to long-lasting competitive advantage.

This is true in some cases, but not all.

A recent survey conducted by McKinsey & Company concluded that a gap of one year or less between first and second entrants into the market is “meaningless.”

Given that Amgen and Sanofi-Aventis are both large pharma companies with considerable experience in taking drugs to market, the ultimate winner is likely the company that executes the best commercial strategy, and both drugs should do very well.

That being said, this blogger personally believes that $67.5 million was well worth the psychological dagger that Sanofi-Aventis was able to put in Amgen’s corporate heart by pulling off the victory at the end.

Amgen got outmaneuvered.

There’s nothing worse than having victory slip through your hands at the last minute!

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Category: Regulatory Affairs
Keywords: PCSK9 Inhibitors, LDL Cholesterol, Praluent, Repatha, Pediatric PRV, Amgen,

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