Please ensure Javascript is enabled for purposes of website accessibility

Here's Why Ionis Pharmaceuticals and Akcea Therapeutics Plummeted Today

By Brian Orelli, PhD - Aug 28, 2018 at 12:51PM

You’re reading a free article with opinions that may differ from The Motley Fool’s Premium Investing Services. Become a Motley Fool member today to get instant access to our top analyst recommendations, in-depth research, investing resources, and more. Learn More

The FDA gives a thumbs down to their triglyceride-lowering drug Waylivra.

What happened

Shares of Akcea Therapeutics (AKCA) are down 26% at 11:41 a.m. EDT while Ionis Pharmaceuticals (IONS 1.44%) is down 16% after the FDA issued a complete response letter -- the agency's euphemism for a rejection -- for their triglyceride-lowering drug Waylivra that was up for approval for a rare genetic disease called familial chylomicronemia syndrome (FCS).

So what

Rejected stamp

Image source: Getty Images.

Akcea Therapeutics and Ionis Pharmaceuticals squeaked though the FDA's Metabolism and Endocrinology Products Advisory Committee with a 12-8 recommendation to approve the drug, with a majority of the outside experts voting that the efficacy justified the risk of thrombocytopenia, or low blood platelet levels, which is associated with taking the drug. As I wrote after the vote, given the large number of dissenting votes, an approval wasn't a slam-dunk since the FDA doesn't have to follow the advice of its outside advisors.

There was also the issue of Akcea and Ionis changing the proposed dosing of the drug and how patients should be monitored for thrombocytopenia compared to what was done in the clinical trial that supported the approval.

Blood vials on a diagnostic order form

Image source: Getty Images.

Now what

Unfortunately investors are in the dark about exactly what Akcea and Ionis have to do to eventually get Waylivra approved. The lack of disclosure isn't atypical as companies often want clarity from the FDA about a path forward before making a public announcement, but it seems likely this won't be a quick fix; it may involve running another clinical trial.

Waylivra is also being tested in a phase 3 trial for patients with familial partial lipodystrophy that is due to read out next year, which might help supplement the application for FCS if the FDA simply wants more data on the risk of thrombocytopenia.

Invest Smarter with The Motley Fool

Join Over 1 Million Premium Members Receiving…

  • New Stock Picks Each Month
  • Detailed Analysis of Companies
  • Model Portfolios
  • Live Streaming During Market Hours
  • And Much More
Get Started Now

Stocks Mentioned

Akcea Therapeutics, Inc. Stock Quote
Akcea Therapeutics, Inc.
AKCA
Ionis Pharmaceuticals, Inc. Stock Quote
Ionis Pharmaceuticals, Inc.
IONS
$36.60 (1.44%) $0.52

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

Related Articles

Motley Fool Returns

Motley Fool Stock Advisor

Market-beating stocks from our award-winning service.

Stock Advisor Returns
345%
 
S&P 500 Returns
119%

Calculated by average return of all stock recommendations since inception of the Stock Advisor service in February of 2002. Returns as of 05/16/2022.

Discounted offers are only available to new members. Stock Advisor list price is $199 per year.

Premium Investing Services

Invest better with The Motley Fool. Get stock recommendations, portfolio guidance, and more from The Motley Fool's premium services.