Crucially, eteplirsen has been assigned Priority Review status - designated to drugs that offer benefit over existing therapies, or provide a treatment where no adequate therapy exists - which means a decision on the file should be reached by February 26 next year.
The company is seeking permission to market the drug for patients with the rare degenerative neuromuscular disorder who are amenable to exon 51 skipping, which equates to around 13% of the total DMD population.
Eteplirsen addresses the underlying cause of DMD by enabling the production of a functional dystrophin protein, and clinical studies have demonstrated a broadly favourable safety and tolerability profile as well as efficacy for the drug, says Sarepta.
This is going to be a great few years going forward....
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