I'm stunned by the comments of the FDA regarding the PhaseII data and the prospective phaseIII trial design. This is pre-AA bureaucratic FDA mindset. Nothing has changed. They have reasserted their power and have sentenced hundreds of 8 yo Boys afflicted with DMD exon 51 that are currently ambulatory and that are not enrolled in the upcoming phaseIII trial a life in a wheelchair. Factoring the phaseII dataset, the AA statute and the advocacy groups passion and meeting with the FDA I really thought that this AA decision was a given. There is something really rotten at the FDA. There was no downside to a [+] AA decision. None. The data supported approval, eteplirsen is extremely safe and it is extremely important for these boys to be administered this drug. If the FDA requires a placebo cohort for the upcoming PhaseIII trial versus age-correlated historical data I will conclude that the FDA needs to be investigated for corruption. How do these families feel after receiving positive, enthusiastic responses from the FDA's point person. Lastly, I would like to apologize to RU/ Pasteur who happened to be correct on his call. Though I agreed with his analysis I thought the FDA was trying to get life saving medicines to patients that needed them via the AA statute. Obviously I was wrong. I apologize. I would also like to apologize to the boys afflicted with DMD and their families for the actions of a corrupt, bureaucratic gov't that doesn't give a #$%$ for it's subjects.
Sentiment: Strong Buy
I am not surprised. Look at the inept congress. Those in power only care about their own and themselves.
If a high ranking member of the FDA had a son with the disease I am sure the decision would have been different. And what about all the disabled veterans the country creates and abandons? This drug should have been made available to as many as possible in the quickest time.
The gutless FDA was so afraid of stem cell treatments it wouldn't allow Geron's phase 1 study to include partial spinal cord injuries, which the Rx was developed for. It insisted on complete spinal cord injuries, because those patients had less to lose if there were safety issues..( the Rx had never worked in animal models of complete spinal cord injuries. Geron showcased movies of its excellent results in rats with partial spinal cord injuries). For the same reason it limited the study to thoracic spine injuries. It refused to allow cervical spine injuries. Geron couldn't adequately enroll the trial because the use of airbags has virtually eliminated thoracic spine injuries, which were mainly caused by steering wheels in crashes. This despite the patients and families begging to be tested, willing to take any risk. After spending an enormous amount of time and money, Geron gave up and dropped its stem cell program. Now there is no treatment for these patients.
Dead on brother! When BIIB & ELN went through this years ago taking " TYSABRI " off the market because of deaths related to the drug. It ruined the lives of all those suffering from MS. In fact, all family members suffered. There's countless drugs on the market right now with worse consequences. Just turn on your TV an and play close attention to the " Side Affects " when running an ad for XYZ drug. Through pressure from the families and the media they where pressured into changing their minds on that stupid call. They sentenced everyone taking TYSABRI to a death sentence. Death, maybe not. Death would've been better then suffering from MS the rest of your life. And knowing you had " Quality Of Life " with the drug, and pain and suffering without? Hopefully the FDA will come to their senses on this call.
BTW: I have no position in SRPT as of now. I did buy ELN, BIIB, And ZIOP when they took a hit of the FDA discussions regarding their primary source of income.
Big difference between BIIB situation Vs Etep. You are comparing oranges w/apples......BIIB did larger studies and several before they were granted marketing approval........after the safety risk, they did more studies just to refine their label to specifically target patients who truly benefits.......so the FDA was right in demanding more studies to fine tune the drug's target population. With 12 patient single center data, you think you know everything about this drug??????
FDA position, despite parent meetings and the new laws, apparently remains:
"... that its better not to let The People try a safe but potentially ineffective drug, if the natural course of the disease MIGHT be better than previously thought. Better to risk inaction and downhill clinical course than allow parents to make their own decisions with a safe drug. Maybe Etiplirsen's reprogramming exons to make dystrophin is meaningless because the natural course of the disease has some questions. Lets take another few years to be sure. What's more important, the risk of more kids in wheelchairs (which can't easily be proven quantitatively) or the reputation of the FDA.
bio - no need to apologize to ru/pasteur - just because he has the same, stunted mindset of the FDA doesn't prove he is "right". There is nothing logical or "right" about what the FDA has done here - this turnaround based on the failure of the Prosensa trial results makes absolutely no sense and goes against all the goals/rules of the AA legislation for rare and deadly diseases. I am the last person to subscribe to conspiracy theories, but something doesn't smell right here. As CG described, they used the Prosensa data, which at best showed inconsistent results (not every treated boy showed an increase in dystrophin and the levels were not consistently high for those that did), to question dystrophin as a surrogate, because the Prosensa treated boys showed declines in their 6MWT, but then turned around and ignored the same fact (i.e. that the boys over age 7 (treated and control) showed declines of over 65 meters in their 6MWT over one year) in assessing the "natural history" of the disease. This is not cherry picking data - this is turd picking the worst possible data to raise doubts.
Agreed. Plus, for some reason, the FDA decided to set up SRPT by suggesting they would positively review an NDA application off of their P2 data, only to turn around, and find fault with everything including 6 mwt, dystrophin, etc. They have created a disease that can't be adequately evaluated with regards to approving an effective drug, because there is no accepted way to measure anything. #$%$? Also, drawing comparisons between two drugs that are given in wildly different doses seems foolish. How can you expect the response to 6 mg/kg to be indicative of what a similar drug might produce at 50mg/kg? Woodcock should be ashamed of the additional pain and anguish her misleading remarks, and parent visits have created. Reprehensible and truly heartless.