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Sarepta Therapeutics’ CEO Chris Garabedian Discusses Orphan Drug Eteplirsen For Duchenne Muscular Dystrophy

December 4, 2012

       On December 3, 2012, Chris Garabedian, President and CEO of Sarepta Therapeutics, was interviewed by Bloomberg Brief Healthcare Finance’s Elizabeth Krutoholow, about orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD). The Q&A Interview can be viewed in a December 3, 2012, Bloomberg Brief Newsletter (BBF).

       Several points from the Q&A session in the Bloomberg Brief Newsletter :

– Eteplirsen “ … only treats 13% of patients but the technology can treat more than 80% of the population. It would take 20 drugs to reach that many patients. We are focusing on the top 5 targets, which will cover about 45% of the exon skipping population …”

– Sarepta Therapeutics is in the process of requesting a meeting with the FDA, which should be during the 1st Q2013 with objectives of aligning “endpoints & design of our confirmatory trial” & to determine if Eteplirsen’s current data can be used to file a NDA under Accelerated Approval

– Discusses interest in an ex-US rights deal.

       Also, Sarepta Therapeutics’ Chris Garabedian participated on December 4, 2012, in the Deutsche Bank dbAcess BioFEST at 7:50 AM.  Please reference’s Transcript

       Several points from the conference call transcript

–  Strong “advocacy voice” of the Duchenne Muscular Dystrophy community for Eteplirsen 

–  Ex-US partner interest for Sarepta’s Eteplirsen and their DMD Pipeline, which “would really be a big catalyst for the company to accelerate the broader program” (DMD pipeline) 

–  Discussion on collaborations that Sarepta has on exon 53, exon 45, and exon 50; “those 3 drugs alone are 3.5x the market opportunity when you take into account we have freedom to operate in Europe … and the US market opportunity”.   

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

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