Kyndrisa a new pristine BMRN drug? No history of problem side effects found searches? Market away your problems BMRN
So any background information on Kyndrisa? Trials info? Testing? Side effects? Nothing? Bury bad facts with new slick Name. Now that is a company I can rely on. One that doesn't mind a little window dressing for marketing. Tampering with media marketing, welcome to the dark side of Pharmacy business.
"Australia court rules against breast cancer gene patent". BBC online NEWS. In short a Discovery of a gene sequence or marker is not patent-able. It is naturally occurring and was not invented, just discovery alone is not.
Drisapersen trials released information in the FDA ADCOM documentations that is rare in the world of Drug Development. A trial this bad and with dangerous adverse effects usually never get to see the light of day. Drug companies bury this type of data. No sense letting competitors and investors knowing the results. Not so in Bio Marin's case. They submitted the trial results to the FDA in spite of failed trials. This FDA documents presented the worst case of a company trying to rangle approval, not unusual by itself. Here we also see the extent of hidden failure as well as attempt to cover adverse effects and lack of meaningful data that drug company will go to extract dollars even it could injure or kill. GSK dropped the drug without releasing data, this I would expect as common current practice of drug companies. To see the extent of a drug failure is eye opening.
BMRN EU patient is on Gene sequence. US disallows discovery as invention. US SRPT argument with BMRN seems to be they claim they own all exon 51 sites by discovery only.
BMRN would have to start massive trials AGAIN with all new locations, IRB approvals and so on. While these kids get BMRN to test their theory AGAIN that Drist works. A Year and a half wasted? Two years? for extended studies? What if it turn up that it still doesn't do squat? FDA with mud on their faces? Families squawk, man o man what a firestorm will be created.
At same time SRPT not accepted because of lack of ??? More data needed on younger or older chair bound kids... What ever, with an FDA delay again it would be a complete nightmare for the FDA. The FDA dropping a effective drug so BMRN can run trials? I don't think so..What do you think?
The DMD community, all they are asking for are SAFE and Effective drugs. So why the surprise when the FDA handed exactly that back to them. Don't send them drugs that could kill (Per FDA x2), so the FDA won't.
Effective why so many drop outs then? Not mentioned during the meeting BTW. I can't imagine a kid in pain doing very well in a walk test., still no mention of how that effected selected patients.
Of topic: Give the FDA a mountain of paperwork to work through. Hours of work/overtime made more difficult having to sift for proof. And you guessed it BRRN are in shock over them seemingly being #$%$ off at them. They got bashed with 1 million pages dropped back on their heads. As in "How do you like them apples".
Shocker!!!! It was the FDA that asked for Natural history data. Hoffman the Twitz and and McDonald's UC Davis mixed data confused the natural history data by lumping it into ALL DMD.
So against 51 data Eteplirsen stands out great. I expect this same type of natural history data to will expose lower DATA results in the other less quick to onset exons. Will that be the next Exon 53,or 43 or 8 Data shocker for shorts,? I can read the headline now, "SRPT less effective" shows data not as effective for other exons as was for EXON 51. (because of natural history being different).
In short, SO with DMD nature history all forms of DMD WILL HAVE DIFFERENT DATA SETS. Shocker!!!!
PS SRPT had to find this out itself since all the money raised an spent by DMD Charities and drug company no one had published a simple study on the natural history of different DMD forms.
Pat Furlong and Debra Miller are selling "HOPE TO FAMILIES" to the FDA. That is their current press. That is their public reasoning showing up and speaking for BMRN. This is false hope. They don't seem to recognise the difference. That is what trials are about....
So yes they are going to hide behind selling "false hope" as logic of support only to BMRN.
We could see kids coming from all over the world for treatment, adding to the numbers in the US. Likely? Ed mentioned offers from wealthy for treatment. We had a story of the British couple here for trials, moved just for treatment.
No way he could charge 1000% more with a high cost to manufacture in the first place. Maybe BMRN will be looking for a new CEO? Good fit don't ya think?
Drisapersen, Glaxo Reported 2013. Reported by the STREET. Patients with thrombocytopenia are at increased risk for uncontrolled bleeding. Proteinuria is the presence of excess protein in urine. Severe proteinuria can be a sign of kidney damage. Kids quietly placed in hospitals.
How can this be ignored by BMRN? They Don't care. Either of these could and will kill. It's a matter of time. All it take is a fall, bump in the head and that's it. Can they cover it up for a while? Accidental causes is how it will be listed by the Loss prevention Lawyers.
Does BMRN Chemistry have a history of adverse events and deaths?
I noticed on the BMRN drisp side adverse effects was many injection site reactions, however no documentation for PAIN at injection site? Or listed separate ? Pain is listed as zero.
How could you have injection site reactions with no (Zero 0) pain at that site? Pain was miss reported in the trial. Zero pain outside of the injection site only was reported? No Pain listed under injection site reported, it's assumed? They continue miss report trial results on Pain. My concern is this, It seems they are reporting injection site reactions but no pain scale documentation in their list of reactions. . . or separated it out. it's a misdirection with reported data.
I would have listed injection site pain as PAIN on a scale of 1-10. EU Medical and Nursing may not use the same Pain reporting as in the US as part of Vital signs.
PS please follow up..
The two removed? They became an important part of the trial with the exception of the walk test. With current studies and those two, new endpoints are being brought forward for non-ambulatory boys. The cardiac and lung function both improved. All this is new Objective data.
Biopsies taken, yes. Surviving, yes. It will take years for the wheelchair kids to reach the final phase of being studied for Cardiac and lung function. SRPT is THE Pioneer DMD drug researcher.
Well Phil Reames the great investment manager. Big on BMRN.. Blow hard...FB
Kyndrisa name change to hide data. Internet searches for Kyndrisa, none? Why it's pristine, nothing wrong with a shell game to hide off comments and Facts about Drisapersen. BMRN marketing people with headwinds from parents could easily say "Drisapersen? Huh, what's that?". Drisapersen no we have "Kyndrisa" it's much better.
Read the briefing Doc,s. Just the ulcers shown is enough to make anyone cringe. Show a family all the reported side effects and the injection sites ulcers before volunteering for dosing. You would have to be stupid to agree to it. I would have to explain the reports of side effects, for early death form many organ systems affected, for months of painful injection sites to resolve, months of treatment for skin ulcers......and on...
They skipped over one other BMRN comment on the Dystrophin essays which was :"Less Drug uptake in more damaged cells". Suggesting older boys will have less uptake, and respond differently. Young kids less damage with better uptake. I found that odd indeed. Does that also require more study or just more drug they can't have.
Drisapersen is toxic, the adverse effects could and will send a few to an early grave. BMRN if not saying a thing. How long before they miss one lab and they die from a bleed, renal failure less likely. Anything I missed? We see from today's news that BARD sell killer devices. Same thing here with Drisapersen, let a few die and clean it up later.