Primary Outcome Measures:
The primary efficacy endpoint will be the change from baseline in the percent of dystrophin positive fibers in muscle biopsy tissue as measured by immunohistochemistry (IHC). [ Time Frame: 20 Weeks ] [ Designated as safety issue: Yes ]
The primary efficacy endpoint will be based on the pre-treatment and post-treament percent of dystrophin positive fibers as measured in the muscle biopsy tissue on(IHC).
Secondary Outcome Measures:
The secondary efficacy endpoints will be the change from baseline in: CD3, CD4, and CD8 lymphocyte counts in muscle biopsy tissue; 6-Minute Walk Test (6-MWT) distance. [ Time Frame: 20,80 Weeks ] [ Designated as safety issue: Yes ]
A key secondary efficacy endpoint will be based on the pre-treatment and post-treatment CD3, CD4, and CD8 lymphocyte counts as measured in the muscle biopsy tissue as well as a 6-Minute Walk Test(6-MWT)distance.
Study Start Date: February 2012
Estimated Study Completion Date: July 2015
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
Drug: AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion over a period of at least 60 minutes. Their are two treatment groups, 30 mg/kg and 50 mg/kg.
February 6, 2014
SRPT Advocates set to meet with Congress on Sarepta DMD drug
An advocacy group, along with a panel of Duchenne’s muscular dystrophy researchers, is set to meet with U.S. lawmakers tomorrow to urge accelerated approval for Sarepta’s (SRPT) eteplirsen. WHAT’S NOTABLE: According to a report in the Boston Business Journal, The Jett Foundation, an advocacy group organized by families affected by Duchenne’s muscular dystrophy, is set to meet with U.S. lawmakers tomorrow to urge accelerated FDA approval for Sarepta’s drug. The Foundation’s government affairs team will also be joined by a panel of Duchenne’s researchers. The Foundation said a similar meeting last year spurred 30 congressman to write the FDA urging fast action on eteplirsen. Sarepta said it is not involved in Friday’s meeting. ANALYST COMMENTARY: In a note to investors earlier today, analysts at RW Baird raised their price target on shares of Sarepta to $35 from $22 following updated data from the company’s Phase IIb trial reported yesterday. The firm said the update on pulmonary data provides incremental evidence of efficacy following the 120-week, 6-minute walk test data reported last month, which showed a general stabilization of walking ability in eteplirsen-treated patients. Baird maintains an Outperform rating on shares of Sarepta.
that is very possible soon.
enroll 36 boys in new trial for exon 51 and after treatment for 36 weeks and the boys follow same path as the past trial outcomes approve the drug based on a natural history change.
real clear and in plain view .....a circle of evil ......will not work
STRONG BUY BOYS...................................................................................................I
Funny how Sarepta passed on the FDA's words and got suits.........they will be at court also as a witness ,so how can everyone get out of this ''approve the drug''
All evidence points to a winner drug.......2 plus 2 does = 4
FDA causes lawsuits
FDA not dragged into suit if approved
FDA ,all statements would be true that caused the lawsuits
FDA has natural history change by drug for approval method
''The drug has great safety''
''The drug simply works,the first in history as well......
one year of exon 51 drug sales could be 3 billion easy and this is early sales most times you get 5 times yearly sales and the other platforms are worth much.....why would you sell for 5 billion later,i sure would not
why waste time on this much proof...........
again the real surrogate endpoint it there '''A change in the natural history of this disease by Sarepta's drug''
Sorry i got busy yesterday...
When seeking early approval for a deadly disease like DMD for children you need nothing more than good safety and proof that you changed the natural history of this disease by extending life and or changed the harsh progression to a much slower progression .It is too many age differences and progression levels to get any other real details .A early approval followed by a collection of information over 10 years is way better than a trial for 10 years for the same information.
February 1, 2014 /
We have a problem, one that I never expected us to face. When we launched Jett Foundation thirteen years ago, I anticipated major hurdles. I thought the science might not be advanced enough to lead to new medicines in time to save Jett’s life. I thought we would have trouble raising enough money to fund crucial medical research and drug development. I worried that Jett might go through an ugly stage and I wouldn’t be able to splash adorable pictures all over our website. But never in a million years did I think we would face this: We have a drug that is safe and effective in a small clinical trial that has lasted more than two years. Some of the boys taking the medicine are even IMPROVING, a word that is never uttered in relation to Duchenne. Yet the drug is only available to the 12 kids in the trial, and it could be YEARS before other kids with Duchenne are allowed access. My kids hate when I use their lingo, but I can’t think of a grown-up phrase that says as well as this: #$%$?
Let’s change this NOW. If you’re in a rush, skip to the last paragraph. If you have 3 minutes, continue here:
Many of you have written about this travesty to the FDA. Now it’s time to step it up a notch. Please ACT TODAY and contact your Congressmen and Senators. On Friday, February 7, at 10:00am, there will be an important briefing on Capitol Hill for Congressional staff on a pending decision by FDA on whether to proceed with Accelerated Approval for treatments for Duchenne Muscular Dystrophy. The briefing will feature a panel of renowned DMD researchers and scientists, as well as our government affairs team, who will brief staffers on how they can help. We need YOUR HELP to ensure your Congressmen and Senators send their health policy staffers to this important briefing. (You do not need to worry about coming yourself as we will have parent representatives there and will update the community directly after the briefing.
A similar briefing held last year
Using the change in natural of dmd as a surrogate endpoint to approval a drug..so many different ages and stages of this disease requires this is the only way to deal with a logical measurement.