We know and the parents know Sarepta's dmd drug works well with no safety problems.The biggest factor is no other drug can be compared to it.
Sarepta Therapeutics, Inc. (SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD). Sarepta has agreed with the Agency to initiate a rolling NDA submission and will submit the non-clinical and CMC components of the NDA by the end of this week. As previously announced, Sarepta plans to submit the final component of the NDA by mid-year 2015.
"We will initiate a rolling NDA submission to facilitate the regulatory review of the NDA,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible.”
Dystrophin data will be in mostly in june/july --ish --bulk size
like i said many don't know what they are even talking about..it is very funny but i like long term investing based on those facts it makes me money..haa
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:
and the only reason the kids walked further was because all of them were very young when the trial first started and sarepta's kids were older ''sarepta's trial blew the doors off their trial.
According to Reuters, GlaxoSmithKline (GSK) will no longer be pursuing approval for the experimental drug drisapersen stating that rights to the drug will be returned to the Dutch company Prosensa.
Drisapersen Fails in Phase III Trial
GSK’s decision came after drisapersen failed to impress investigators in a phase 3 clinical trial testing the drug as a possible treatment for Duchenne muscular dystrophy.
While earlier trials indicated that muscular dystrophy patients were able to walk a greater distance over a six minute period, September’s final-stage test run showed failed to show significant improvements when compared to a placebo.
GSK’s return of drisapersen to Prosensa marked the end of a four year collaboration between the two pharmaceutical companies which began in 2009.
Side Effects of Drisapersen
Multiple adverse events were linked to drisapersen during clinical trials during phase 2 and phase 3 clnical trials. – most common were low blood platelet counts and proteinuria.
Blood platelets play a role in blood clotting and low platelet counts, or thrombocytopenia, can result in spontaneous and/or excessive bleeding – such bleeding can be fatal if not treated.
Several patients were also hospitalized after suffering from severe proteinuria (excess protein in the urine). Proteinuria can lead to kidney toxicity and renal injury.
Any parent would switch their kids over to sarepta if drisa was approved ..what parent in their right mind would keep the kid on drisa.
a target of 120.00 Feb.2016 this is what i come up with,,,,,,,,fyi just my own findings..do you own dd
IF YOU REALLY HAD DONE YOUR RESEARCH YOU WOULD KNOW THAT WAS FOOLISH TO SAY.
Sep 23, 2013 - Last Friday, an early top-line Phase III data release for Duchenne Muscular ... of drisapersen is GlaxoSmithKline (GSK) - and fortunately for GSK ... In addition to this, we note that the toxicity remains a serious issue
Comparison or competition ...sarepta is the only real drug option for dmd worth while and shame on biomarin for even trying to long term dose with their drug/
It is always good not to argue with the boss.It is always wise to stop all the dmd parent involvements while meetings are being done...fyi
Most of them do not have a real clue on the price target '' a few here have 100 times more knowledge than any of the analyst do. Just watch the next 6 months here it will tell all