Credit: Shutterstock photoIsis Pharmaceuticals, Inc. 's ISIS subsidiary, Akcea Therapeutics, announced that the FDA has granted orphan drug designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients suffering from familial chylomicronemia syndrome (FCS).
We note that orphan drug designation is granted to drugs capable of treating diseases that affect less than 200,000 people in the U.S. The status makes volanesorsen eligible for seven years of marketing exclusivity in the U.S., following approval for FCS. The granting of the designation to volanesorsen also makes the company eligible for certain other benefits, including tax credits related to clinical trial expenses, exemption from the FDA-user fee and assistance from the FDA in clinical trial design.
Currently, volanesorsen is being evaluated in a multi-center, randomized, double-blind, placebo-controlled phase III study in patients with FCS. In addition, the company intends to begin a second phase III study on volanesorsen in patients suffering from familial partial lipodystrophy later this year.
We are encouraged by the FDA granting orphan drug status to volanesorsen. Given that an estimated one to two persons out of a million suffer from this disease worldwide, there is a strong unmet need for drugs for the treatment of FCS.
Apart from volanesorsen, other lipid disorder candidates in the company's pipeline include ISIS-APO(a)Rx (phase II - cardiovascular disease) and ISIS-ANGPTL3Rx (phase I - mixed dyslipidemias).
We note that Isis Pharma's only approved product, Kynamro, is a lipid-lowering drug approved in the U.S. for cholesterol management in patients suffering from homozygous familial hypercholesterolemia. Isis Pharma has an agreement with Genzyme, a Sanofi company SNY , for marketing the product in the U.S.
We expect investor focus to remain on pipeline updates from the company.
Isis Pharma currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Achillion Pharmaceuticals, Inc. ACHN , AMAG Pharmaceuticals, Inc. AMAG and Sanofi. While Achillion and AMAG are Zacks Rank #1 (Strong Buy) stocks, Sanofi holds a Zacks Rank #2 (Buy).
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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
