. . . could no longer hold srpt as a lottery ticket – with its current long-shot status – when so many other more-promising, lower risk biotechs were being given away . . . didn’t get the best prices on my sells, but did get very near the lows on my buys . . . all in all, a good day . . . so best of luck to those of you still in the game and . . . adios . . .
. . . as I pointed out before, i2 . . . according the the FDA, "Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product (i.e., one that has not been approved by FDA). FDA is committed to increasing awareness of and knowledge about its expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices. . . . Wherever possible, use of an investigational medical product by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access." . . .
"It took me a couple of days to get over my anger" . . . at "bureaucratic, last in their medschool graduate, psuedoscientists"? . . . yeah, you're the voice of reason now . . .
. . . I would expect that most everything in the Eteplirsen Briefing Document (NDA 206488) will be highly positive in its spin, since it's written and submitted by sarepta . . . you, q7, made it sound as if it were the FDA lauding the drug's performance, when, in fact, they appeared strongly negative on the ambulation comparisons in their own briefing documents . . .
. . . why approve etep on such incomplete data, td, when "expanded access" is available from the FDA? . . . more patients could receive the drug until it's proven to work or not . . . plus, the taxpayers/insurance-premium payers wouldn't be ripped off for hundreds of millions if the drug is proven to be useless -- as the FDA's current analysis seems to indicate . . .
. . . yup, the more I think about it, the more it seems a perfect fit for etep at this stage . . . according the the FDA, "Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product (i.e., one that has not been approved by FDA). FDA is committed to increasing awareness of and knowledge about its expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices. . . . Wherever possible, use of an investigational medical product by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access." . . .
. . . until the current trials show definitive results . . . according to the FDA briefing document, the agency seems to strongly suggest that the performance profiles of these patients -- sadly -- is within the typical progression profile for this disease . . . “Figure 2 shows the change over time in NSAA scores for all 12 eteplirsen-treated patients in Study 201/202. All show progressive declines in NSAA scores, with six patients moving to NSAA scores that have been reported to be associated with being within one year of loss of ambulation, and an additional four patients moving to scores associated with being within 2 years from loss of ambulation.” . . . “Figure 3 shows the change over time in rise time from the floor for the eteplirsen-treated patients in Study 201/202. Most had marked increases in rise time and several became unable or nearly unable to rise from the floor, which predicts a high likelihood of loss of ambulation within 1 or 2 years." . . . so in a year or so, we’ll likely know the truth: that etep works – for whatever reason – or it doesn’t . . .
. . . since you haven't provided a page number from the briefing docs, q7, I'm going to assume for now that your statement -- "Per the FDA briefing docs... these kids should be in wheelchairs" -- is merely your own personal view/wishful thinking . . . fair enough . . . everyone is entitled to an opinion . . . but, from what I read, your opinion is not shared by the folks at the FDA who will be making the final decision . . .
. . . "Per the FDA briefing docs... these kids should be in wheelchairs." . . . really? . . . on what page can I find that? . . . thanks . . .
. . . send your "hard evidence" to Adam Feuerstein . . . if what you say is both true and, more importantly, material to the etep defense, he might publish an update for Monday night or early Tuesday that could quickly move the stock price higher when U.S. markets re-open . . . however, if it's just more message board baloney, don't bother . . . good luck . . .
I didn’t mean to imply, o7, that all animal testing is without merit . . . for that matter, check out research at Duke University and a January 2015 article published by the National Institutes of Health (NIH): “Gene editing improves muscle in mice with muscular dystrophy” . . . “Three teams independently used the CRISPR/Cas9 gene-editing system to restore expression of the gene responsible for Duchenne muscular dystrophy in mouse models.” . . . but if you favor canine results: “Gene therapy treats muscular dystrophy in dogs" (November 2015) . . . “A team led by Dr. Dongsheng Duan from the University of Missouri School of Medicine has been working on DMD gene therapy for years. The group had previously developed a miniature version of the dystrophin gene, known as a microgene, and achieved successful gene therapy in dystrophic mice. In the current study, they set out to examine the feasibility and safety of the approach in larger animals. The research was funded in part by NIH’s National Heart, Lung, and Blood Institute (NHLBI). The study was published on October 15, 2015, in Human Molecular Genetics.” . . .
. . . patently false? . . . hardly, o2 . . . the fact that the FDA felt compelled to call an advisory committee on etep in the first place and then to provide comprehensive briefing documents questioning the very validity of its results tells me -- and anyone else with even a modicum of common sense -- that they have serious doubts about the "perceived" efficacy of this drug . . .
. . . would agree, o7, that animal drug studies can provide sound scientific results that can be replicated -- in other animal studies . . . unfortunately, countless drugs that have generated positive outcomes in animal studies have later failed to provide a meaningful benefit in humans . . . that said, newer studies -- employing genetically engineered animals -- appear to be providing more useful data . . . the fundamental problem for etep, as i see it, is that much of its benefit seems to rely on a positive interpretation (spin) of circumstantial evidence . . . had there been robust positive data from the start, srpt would likely have had a partner early on, and etep would already be on the market . . .
. . . according to wiki: "The placebo effect is a pervasive phenomenon; in fact, it is part of the response to any active medical intervention. The placebo effect points to the importance of perception and the brain's role in physical health. Placebos can produce some objective physiological changes, such as changes in heart rate, blood pressure, and chemical activity in the brain, in cases involving pain, depression, anxiety, fatigue, and some symptoms of Parkinson’s." . . .
. . . you're right on that last point, o7 . . . unfortunately, that FDA conclusion is likely to come in the form of a CRL . . . we'll see . . .