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Sarepta Therapeutics, Orphan Drug Eteplirsen, And The J.P. Morgan Healthcare Conference

January 10, 2013

       On Wednesday, January 9, 2013, 4:30 PM PST/7:30 PM EST, Sarepta Therapeutics gives a presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation is given by Sarepta Therapeutics’ President & CEO, Chris Garabedian.

Here are a few highlights from the presentation for orphan drug Eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD) :

1) DMD facts :

– Approximately 35,000 boys worldwide; affects approximately 1 in 3,500 boys worldwide

– Eteplirsen has potential of benefitting > 4,000 or 13% of patient population in US & EU

– 28,000 or 80% of DMD patients amendable to Exon-Skipping

2) Eteplirsen facts :

– Mid-scale production in 1st half of 2013

– Presented Study 202 Phase IIB Extension Study for 48-weeks

– Presented 6MWT 62-week data

– Requesting end of Phase II FDA meeting 1st Q2013

– Planning initiation of Confirmatory Study by end of 2013 with dosing in 1st Q2014

3) FDA Safety & Innovation Act (FDASIA/PDUFA V) legislation enacted July 2012 offers new regulations for rare diseases and orphan drugs that can be used for Eteplirsen for DMD :

– Section 901: Expanded Accelerated Approval (AA) – per Adam Feuerstein’s live tweets, Eteplirsen is good candidate for this

– Section 902: Breakthrough Therapies

4) Preparing for EOP2 Clinical Meeting with FDA in 1Q 2013

5) Discussion of ultra-orphan drugs :

*** – Page 28 Chart identifying prices of ultra-orphan drugs with average price of $350,000

*** – Page 29 showing the potential commercial opportunity in US & EU :

– 1,950 patients in US with $0.6 – $1.0 billion market

– 2,600 patients in EU

*** 6) Page 30 identification of 2013 activities & milestones

7) Per Adam Feuerstein’s live tweets in breakout after presentation :

– Sarepta’s closest competitor Prosensa holds patent on exon 51 in EU

– FDASIA’s breakthrough designation process is 60-day process, so can do it if warranted; already getting responsiveness from FDA without breakthrough designation.

References January 9, 2013 article titled, “Will Sarepta Be Bought Out in 2013 ?” + Video

Orphan Druganaut Blog Post titled, “Review of Phase II & III Orphan Drug Clinical trials for Duchenne Muscular Dystrophy: Journal Article”

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


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