Question...Do you see any data from Biomarin about dystrophin production at all? It is the only real reason for keeping these kids walking...Why would you approve a drug that has no benefits that matter?
1 Not yet recruiting Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients Condition: Duchenne Muscular Dystrophy
Interventions: Drug: SRP-4045; Drug: Placebo
Would Biomarin have the money or stock power to buy Sarepta for 5 billion or more ,that is the only real hope for Biomarin..
The FDA would be foolish to give approval to Biomarin for a drug that does nothing but harm..I hope the hell they can read
Sarepta has a mountain of evidence that it does in fact work,,
this is very plain to see if you look.................
The pediatric rare disease PRV program was modeled after the NTD voucher program, which is still up and running and is still available. The main intent of that program was to provide an additional incentive for drug sponsors to pursue these drugs. What both of them have in common is that if you have a new product that qualifies -- in this case for a rare pediatric disease -- and the marketing application is submitted to the FDA, and it qualifies for a priority review, then if that drug is approved, at the time of approval a voucher will be given to the drug sponsor. They can take that voucher and apply it to a different drug in the future to convert that marketing application review from a standard review to a priority review. The main difference between a priority review and a standard reviews is that the FDA's review clock is 4 months shorter for a priority review.
Another benefit is that the drug company doesn't have to use that drug voucher themselves. They can sell it to someone else, and that can be a financial incentive. That is the very basic outline of the voucher program. What does the pediatric voucher program have that is different from the NTD voucher program? The pediatric voucher can be transferred any number of times. There is no limit to how many times the voucher can be sold and resold and resold again, which we hope will make it more valuable and more flexible. The NTD voucher can only be transferred once. The sponsors also have to notify the FDA when they want to redeem the voucher; for the pediatric voucher, it's 90 days in advance of the marketing application, whereas for the NTD voucher, it was a year. This was an attempt to provide more flexibility and increase the voucher's value.
What the pediatric voucher also offers is the opportunity to designate the product during the development time, during the investigational new drug phase. It can be designated as a rare pediatric disease if the sponsor chooses. They can do this if they want to. I
250 mil will get us any where.................worth 5 times more
The magic number before the next climb is about 150 pts lower or about..maybe less
many small caps never recovered from last year....The Fed really knows how to cause sell offs and get tax monies in.... long term always pays off in the end.
AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 Weeks Compared to Placebo in Phase IIb Trial for the Treatment of Duchenne Muscular Dystrophy
BOTHELL, WA, Apr 02, 2012 (MARKETWIRE via COMTEX) --AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that treatment with eteplirsen met the primary efficacy endpoint in a randomized, double-blind, placebo-controlled Phase IIb study in boys with Duchenne muscular dystrophy (DMD). Eteplirsen administered once weekly at 30mg/kg over 24 weeks resulted in a statistically significant (p ≤ 0.002) increase in novel dystrophin (22.5% dystrophin-positive fibers as a percentage of normal) compared to no increase in the placebo group.
it gets better @36 and 48 week but 24 is the key starting point
much higher production
Estimated Enrollment: 160
Study Start Date: September 2014
Estimated Study Completion Date: August 2016
Estimated Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treated Group
Approximately 80 patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to correction by exon 51 skipping. Drug: eteplirsen injection
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 48 weeks.
No Intervention: Untreated Group
Approximately 80 DMD patients not amenable to exon 51 skipping
news anytime after close or during this week also very possible??
I just know long term will be freaking outstanding.......................