New Mega-Blockbuster Cholesterol Drug Class Faces Make-or-Break FDA Panels

A U.S. Food and Drug Administration (FDA) advisory panel is expected to meet on June 9 to consider the drug application of alirocumab from Regeneron Pharmaceuticals Inc. (NASDAQ: REGN) and Sanofi (NYSE: SNY). Amgen Inc. (NASDAQ: AMGN) also has a FDA advisory committee slated for June 10 for Repatha (evolocumab). Investors will also have to consider what this means for Pfizer Inc. (NYSE: PFE) with its PCSK9 trial underway.

For Regeneron and Sanofi, alirocumab is being evaluated to manage LDL cholesterol. This also includes people who do not manage to get their target LDL levels down by using statin medicines alone. Regeneron is developing alirocumab as part of its global antibody in collaboration with Sanofi. Alirocumab is a fully human monoclonal antibody that binds a protein called PCSK9. What matters here is that the PCSK9 inhibitors are a new class of drugs to fight cholesterol.

On Amgen, the FDA advisory committee will discuss the safety and efficacy of biologics license application for injection as adjunct to diet to reduce LDL and total cholesterol, as well as triglycerides. Amgen’s PCSK9 class of drug is being evaluated as either a combination with a statin or statin with other lipid-lowering therapies, or as a standalone agent in combination with other lipid-lowering therapies in patients aged 12 and above with homozygous familial hypercholesterolemia.

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Pfizer recently announced that it was launching competitive grants to advance cardiovascular disease research. Its drug candidate is bococizumab, an investigational PCSK9 inhibitor, which is currently in a Phase 3 clinical trial program. As a reminder, Pfizer’s Lipitor was the mega-blockbuster statin drug of choice for years with millions of patients using it, and it is off of patent protection and has generics now.

What matters on both PCSK9 drugs up for advisory committee reviews this week is that there is instant mega-blockbuster potential here. In short, annual sales could be well into the billions. There are as many as 15 million potential patients who could benefit from this new class of drugs.

The approval and financial risk here is not just at the FDA level. Insurers already have warned that the costs are exponentially higher than the already-approved drug classes like statins and other alternatives. Another ongoing issue, which is often harder to prove or disprove, is that lowering LDL and other counts actually lowers the risks of a heart attack.

Regeneron has seen its shares rise handily, by 70% or so in the last year. At almost $540 now, Regeneron trades at all-time highs, with a $55 billion market cap. It is also trading above the consensus analyst target price of about $507.

Amgen is trading around $157, shy of its all-time high of $173.60, and even further below its consensus analyst target price of almost $179.50. Amgen’s market cap is just a tad under $120 billion.

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One last consideration here creates another wild card for biotech and pharma investors — and for patients who need and want the new drugs. Formal FDA approval decisions do not always follow the advice and recommendations of the FDA advisory panels.