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Week of 03/04/13 Orphan Drug Events

March 4, 2013

There are no scheduled FDA PDUFA decisions for this week. There are two important earning reports that should be closely watched.

I – Sarepta Therapeutics’ 4th Quarter & Full-Year 2012 Financial Results

This week will be very busy for Sarepta Therapeutics as the company is involved in two important events. Investors are interested in the financials of the company of course, but what everyone is waiting for is to hear what Chris Garabedian, President and CEO, says on the topic of a possible accelerated approval for orphan drug Eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD).

On Monday, March 4, 2013, at 2:50 PM ET, Sarepta Therapeutics will give a live webcast presentation at the Cowen Health Care Conference.

On March 7, 2013, Sarepta Therapeutics will report the company’s fourth quarter and full-year 2012 financial results. At 8:00 AM ET, Sarepta Therapeutics’ Preident and CEO, Chris Garabedian, will host a conference call to discuss the financial results and to also provide a corporate update.

Mr. Garabedian is scheduled to speak on April 10, 2013 at the World Orphan Drug Congress USA, on the topic of leveraging a personalized medicine platform to treat genetic sub-groups.

II – Aegerion Pharmaceuticals’ 4th Quarter & Full-Year 2012 Financial Results

On Wednesday, March 6, 2013, at 8:30 AM ET, Aegerion Pharmaceuticals will host a conference call and live audio webcast to present a business update of the company and to discuss fourth quarter and year end 2012 financial results.

Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide), an oral once-a-day treatment for Homozygous Familial Hypercholesterolemia (HoFH), beat competitor Genzyme and Isis Pharmaceutials’ orphan drug Kynamro (Mipomersen) to market. Juxtapid’s price of $235,000-$295,000/year is much higher than Kynamro’s $176,000/year. It will be interesting to see if Aegerion Pharmaceuticals’ pricing and launch strategy changes due to the competitor’s much lower price.

Please Note: “Symbol List” By Mysid [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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