A Clear Drug-Development Path -- Times 2

Isis Pharmaceuticals (Nasdaq: ISIS  ) and Genzyme (Nasdaq: GENZ  ) have a clear path forward for their cholesterol drug mipomersen. Unfortunately, the Food and Drug Administration threw a fork in the road, and the companies will need to take two routes to a marketing approval.

Mipomersen has been tested in both homozygous familial hypercholesterolemia (HoFH) -- patients with two bad copies of genes that cause extremely high cholesterol levels -- and heterozygous familial hypercholesterolemia (HeFH), or those who have only one bad gene. The HoFH patients, who are fairly rare, are much sicker and usually end up with clogged arteries that result in heart attacks early in life.

Fortunately for Isis and Genzyme, there are a lot more HeFH patients than HoFH patients. Unfortunately, the larger potential treatment group means the FDA wants more data on mipomersen before it'll approve the drug for HeFH patients. The duo won't be able to file until after they run a 12-month trial in HeFH patients. The data for the HoFH patients looks like it's sufficient, although now that they're breaking up the two indications, it may take until the second half of the year to turn in the marketing application to the FDA.

Normally, one approval might be enough to gain some sales in the second indication. Doctors are allowed to prescribe drugs off label and might be willing to take the risk of prescribing it to HeFH patients even without the 12-month data. But given the small HoFH patient population, it seems likely that the companies will charge a fortune for mipomersen, which could limit reimbursement for off-label use.

Given the less-than-stellar side-effect profile and the fact that it's injected, I doubt mipomersen will ever compete against megablockbuster cholesterol drugs such as Pfizer's (NYSE: PFE  ) Lipitor or Merck's (NYSE: MRK  ) Vytorin. But it doesn't really have to; going after the FH patients who can't get their cholesterol under control with the current offerings should be enough to produce decent sales.

Investors just need to wait for Isis and Genzyme to finish their drug-development stroll down the FDA's newly cut path.

Morgan Housel points to three predictions for a wild 2011.

Pfizer is a Motley Fool Inside Value recommendation. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors.

Fool contributor Brian Orelli, Ph.D., doesn't own shares of any company mentioned in this article. The Fool has a disclosure policy.


Read/Post Comments (0) | Recommend This Article (3)

Comments from our Foolish Readers

Help us keep this a respectfully Foolish area! This is a place for our readers to discuss, debate, and learn more about the Foolish investing topic you read about above. Help us keep it clean and safe. If you believe a comment is abusive or otherwise violates our Fool's Rules, please report it via the Report this Comment Report this Comment icon found on every comment.

Be the first one to comment on this article.

Sponsored Links

Leaked: Apple's Next Smart Device
(Warning, it may shock you)
The secret is out... experts are predicting 458 million of these types of devices will be sold per year. 1 hyper-growth company stands to rake in maximum profit - and it's NOT Apple. Show me Apple's new smart gizmo!

DocumentId: 1420790, ~/Articles/ArticleHandler.aspx, 11/26/2014 6:11:43 PM

Report This Comment

Use this area to report a comment that you believe is in violation of the community guidelines. Our team will review the entry and take any appropriate action.

Sending report...


Advertisement