just look at Biomarin's dmd drug efficiently ,safety and dystrophin production and then compare to Sarepta's '' that is the only way to be real.. I think 2/3 of analyst and investors are talking and running stupid.
The goals of the NDA are to provide enough information to permit FDA reviewer to reach the following key decisions:
•Whether the drug is safe and effective in its proposed use(s), and whether the benefits of the drug outweigh the risks.
•Whether the drug's proposed labeling (package insert) is appropriate, and what it should contain.
•Whether the methods used in manufacturing the drug and the controls used to maintain the drug's quality are adequate to preserve the drug's identity, strength, quality, and purity.
The documentation required in an NDA is supposed to tell the drug's whole story, including what happened during the clinical tests, what the ingredients of the drug are, the results of the animal studies, how the drug behaves in the body, and how it is manufactured, processed and packaged. The following resources provide summaries on NDA content, format, and classification, plus the NDA review process
A drug that receives Fast Track designation is eligible for some or all of the following:
More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
More frequent written correspondence from FDA about such things as the design of the proposed clinical trials
Accelerated Approval or priority review if the requisite criteria are met. Accelerated approval is meant for drugs that demonstrate an effect on a surrogate, or intermediate endpoint reasonably likely to predict clinical benefit. Priority review shortens the FDA review process for a new drug from ten months to six months, and is appropriate for drugs that demonstrate significant improvements in both safety and effectiveness of an existing therapy. A fast track application is automatically considered for both of these designations.
Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA
The FDA and the EMA granted etelplirsen orphan drug status in November 2007 and December 2008, respectively. The FDA also granted eteplirsen fast track status in December 2007.
Do you guys honestly think pumping drisa and lining your pockets and being part of these kids permanently losing their health, that they will never likely get back if they get on Drisa drug. This is what i am afraid of and some will be apart of this. DMD is a very bad disease ,
some short term profit takers ,just wait and see.
Now with what a good parent knows now about both drugs/ where would you put your kid? What Trial?
I heard Ed Kaye say Sarepta was ahead in recruiting..
#1 looks like they are after very you kids again ''ones that are not expecting decline''
# 2 where is that Dystrophin outcome measuring located?
3# Are they having trouble getting subjects ?
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A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2015 by BioMarin Nederland BV
BioMarin Nederland BV
Information provided by (Responsible Party):
BioMarin Nederland BVClinicalTrials.gov Identifier:
First received: February 28, 2013
Last updated: March 3, 2015
Last verified: March 2015
History of Changes
If junk gets approved ,Sarepta is a shoe in and will take all the dmd market share in dmd because it works and drisa just does not. very true fact.