I love the idea but it would never happen. No way the FDA would ever allow a human element into a clinical review, known for being unfeeling and filled with Adcom members who put their own careers ahead of patients needs.
Not true at all. 100% of the boys treated with SRPTs Eteplirsen are still walking. That drug is bothe safe and effective.
Zero side effects, zero adverse events.
Sure the patent win for BMRN means many young boys will die, but who cares. As long as business wins out, that is a sacrifice we are willing to make!
It also doesn't matter that the whole thing will be overturned on appeal and the net effect will just delay dying boys from getting life saving medication. The point is everybody at BMRN knows Drisapersen doesn't work and causes liver toxicity, but who cares, as long as shareholders make MONEY!!!
please reply to this message if you believe little boys should die so you can achieve profits you couldn't get otherwise since the BMRN drug can't compete.
I've got some news for you, you were wrong.
Now before you get offended, hear me out, because you'll realize I'm actually giving you a compliment - a well deserved one at that. From the posts I've read of yours, I've become a big fan of yours. You are usually right in what you say, so on the rare occasion you are wrong, that is a new thing, therefore "news".
I typically bypass the turkeys and morons, putting them on ignore, because of their hate filled junk that makes them look like short selling bashers.
I do seek out your posts and a few others because they are typically informative, useful, and insightful, and for that, I say thank you.
Now what news were you expecting today? And do you think that any meaningful news would/could/should be released after market close tonight?
Ah, see, I can help you understand here. People are stupid and they bought BioMarine because of hurricane Joaquin and they thought it would help biomarine stocks ...
Now it appears there will be a vote of no on Drisapersen based on the harsh FDA comments released today. I used to think the FDA would approve both Drisapersen and Eteplirsen but no longer.
At least SRPT is better than TTPH that I've been watching (too risky to buy). After hours it lost 80% of its value as it didn't meet the primary endpoint of the Clinical Trial.
How did BMRN avoid a collapse when Drisapersen didn't meet its primary endpoint?
Actually, it didn't fail. While some can claim that it failed because they defined the wrong primary endpoint from the beginning (specifically the target patient population) that was because they likely didn't know which patients it would be most effective in and which it would be least effective in until they did the trials.
Now that the trials have completed they were able to identify a very large portion of the patient population that the drug has extremely high efficacy rates (specifically those treated early before the disease has progressed past the point of no return). They did it right, casting the net as wide as they could, including all patients, and now they have been able to define the scope of patients that the drug works most effectively with. So with that view, the drug works exceptionally well.
If you are a patient in need of the medicine, you would agree that it works. If you were a short seller or against the company (and nothing wrong with that by the way, lots of shorts make tons of money, as the biotech market specifically is highly manipulated as you pointed out) then you would claim they tried to prove "X" and didn't. While that is understandable, all they have to do is change the endpoint to "Y" and they sail through EU and US-FDA approvals. That is why it shot up today and that is why it will shoot up steadily through the end of the year.
For those that were short, I would cover at this point because very little will take it down from here. You shorts probably made a ton anyway as it was $40 a very short time ago, so probably still in the money.
If they are dumb enough to short ISIS did you really expect smart comments from them?
Posted from BMRN board: As AF points out, the vote is not even a traditional yes or no on efficacy and safety (two separate votes) - instead they are asking them to vote on whether the 3 separate trials either strengthen, weaken or have no effect on approval and then a separate vote on whether safety strengthens, weakens or has no effect on approval - this is a very unique format and completely dismisses the pooled analysis that BMRN portrayed in today's press release. Given the FDA's issues and conclusions on the separate trials and the even worse assessment of safety, it is hard to imagine many "strengthen" votes?
Is this right?
Worse than that, I just hocked pearsby's wife to get money to buy SRPT shares.
Now, how much can I buy for 25-cents?
Aw, c'mon, NOBODY is dumb enough to buy that piece of junk. Come ON. Their shareholders spam other message boards BEGGING people to buy it. They can't get anybody to invest in it, so they try to trick people into it instead.
Stop it. Quit buying dammit. I need to buy more tomorrow and want to get it at today's prices.
Didn't you read the headline? They missed the primary endpoint. Don't look at the underlying data, stick to the headline. Stop thinking for yourself. Just read th headline. Ignore the massively overwhelming positive data for early and mid-stage patients plus the perfect safety profile.
I want mmmoooorrrrreeeeeee.
Only late stage patients where they had already lost ambulatory function didn't meet the end point. For those early and mid-stage patients, they showed very strong results. So, this means that early treatment is essential for success, which bodes extremely well for the US FDA approval as well as in the EU.
Safety was same as placebo, so no safety issues either, makes it a compelling approval case.
It will get approved. They do need to modify the ITT population, but when the file the NDA they will simply update the scope of the ITT group and they are home free. The P3 trials fully supports this in every way and the safety profile is impeccable.
With no other treatments on the market the only change they need to make is the ITT, which only would stipulate early treatment produces maximum efficacy.
Actually, PTCT has a different approach than SRPT and BMRN who are both taking exon skipping approaches.
Translarna is for use in patients whose disease is due to the presence of certain defects (called nonsense mutations) in the dystrophin gene which prematurely stop the production of a normal dystrophin protein, leading to a shortened dystrophin protein that does not function properly.
Sarepta Therapeutics’ eteplirsen and BioMarin/Prosensa Therapeutics’ drisapersen are antisense oligonucleotides (AONs) that bypass a cluster of DMD mutations (specifically, exon 51), and are commonly referred to as exon-skipping therapies.
So they are really targeting different patient groups with different approaches.
Bugs me when management puts their own needs ahead of shareholders.
As of July 28th, there are 106 DMD patients on commercial therapy, including patients from both direct commercial sales and reimbursed early access programs. New countries recently added include Denmark, Norway, and Brazil. Translarna received marketing authorization from the European Medicines Agency in August 2014 for the treatment of nonsense mutation Duchene muscular dystrophy in ambulatory patients aged 5 and over, representing the first-ever treatment approved for the underlying cause of the disease.
Top-line data from Phase 3 ACT DMD expected in the fourth quarter of 2015. In December 2014, PTC began submitting a rolling new drug application (NDA) to the FDA for the approval of Translarna in nonsense mutation DMD. Top-line data from the company's ongoing Phase 3 ACT DMD trial is expected in the fourth quarter of this year. PTC anticipates a potential US approval and commercial launch of Translarna for nonsense mutation DMD in the first half of 2016.
If you make money, she hates you equally with anybody else who is self sufficient or profitable.
She wants everybody dependent on the government. It is the only way she can become dictator.