VRTX has not disclosed what additional data FDA has requested. Given the small patient numbers it will be impossible to conduct meaningful classical phase III studies. So I assume it is something else that is missing, especially since VRTX has stated that FDA is willing to consider approval for sNDA for orcambi without phase III data, unlike European authorities that insist on phase III for orcambi sNDA Less
Verity. Thanks for your thoughtful and insightful reply. I realize how very frustrating delays like this are to you and the CF community affected by these decisions. Obviously, the FDA has to be cautious with young children, but the safety of Ivacaftor in the pediatric population is well established and giving the drug a conditional approval requiring clinical phase 4 data to document efficacy over a longer time period in all 23 residual function mutations would give the proof the FDA is demanding and save lives at the same time. Maybe the White House advisers with an interest in 'personalized medicine' could be asked to have the FDA submit this idea to an FDA advisory committee to allow input from experts and the CF community on the idea of an expedited conditional approval for this sub-population of CF patients likely to benefit?
For some reason the CFF seems restrained if pushing for Kalydeco/Orcambi's use off-label or expanding label. Personally, I think they feel the backlash from the $$$ they got in selling rights, which is ridiculous--would it be better if they just gave the money away or invest in finding more cures! Of the various patients/family, I think abut 1/3 of CFers blame the "big bad pharmaceutical companies" for the price of the meds (as opposed to being thankful they charge so much since it brought all the other competitors out of the woodwork and will lead to better drugs faster). And most of the other 2/3 don't follow the details. And for the few that do, there's not much we can do if the frickin' FDA won't even call a committee and seek public input. Personally, I'm furious: There was no reason to reject especially without the committee review and this will literally cost people their lives.
On a separate note, it looks like any potential competition - even though far, far way - will not get any kind of expedited approval. So this decision - while harming patients (shame on you FDA) may actually be better for Vertex financials long term.
VRTX had the Kalydeco sales guidance for 2016 at $670-$690M worldwide which is only $50M more than 2015, representing only about 200 additional patients. This suggests that expansion to the residual function mutations (there are 3000 ww) was not expected to have a significant impact on revenue this year. There are 200 Kalydeco-responsive patients who are coming off from the 661/Kalydeco trial alone.
Verity- Are CF advocacy groups not expressing their concern to congressional representatives with FDA oversight over this FDA decision regarding the rejection of the sNDA to treat residual function mutations with Ivacaftor?
The sad part is that these residual function CF patients' health will deteriorate further waiting even though the efficacy and safety in the use of this drug has been proven. The FDA's bureaucracy is now demonstrating that 'fast track' and 'breakthrough' designation status offers little in terms of expediting approval. Ivacaftor was the first drug to get this designation by the FDA. Seems to me the FDA is clearly not making this designation a path for expedited approval, as was intended by the law that by creating this designation, was mandating streamlining the approval process for a drug that makes such a difference in the lives of patients.
The trial on which the expansion was based was very small -- 24 people participated and 21 people in the open label. Efficacy was good. It is most likely that the FDA wants to see repeat of the same trial with a larger number of participants.
It was an 8 wk trial. Since Vertex can spend more money now than before, they can run a trial quickly and get the expansion approval in a year or two latest.
there is not enough information on the reasons for the cal to understand the reasoning of FDA. there are 23 CFTR mutations and a total of 1.500 patients, so just about 65 patients per mutation. Does FDA expect VRTX to conduct phase III trials based on such low patient numbers? 65 patients per trial with 30 on placebo and 35 on kalydeco?
safety of kalydeco is proven, preclinical data indicate effectiveness, affected kids might try kalydeco without safety risks and will continue treatment only when effective. so why not give them that chance, they don't have alternatives.
Gilead doesn't have to spend much to buy several biotechs right now. Gilead earns at an annual rate of more than 18 B a year with a 30+ B revenue.
pretty simplistic, I can think of hundreds of reason to distrust politicians, and while I think BO is #$%$, he's only a little below my view of all of them.
HRC told the families of the Benghazi dead that they died bcse of a video? And people say she has experience? In whgat, lies and deception?
This is why you can't trust politicians. Obama wants to look like he cares about patients and pushes the Precision medicine initiative. Then identifies Vertex as the poster child for this new evaluation. Encouraged by this Vertex submits. And then gets slapped in the face. Meanwhile, patients die. But politicians got what they wanted, they appeared to care. That's enough.
Vertex didn't include patients having all of those mutations in their trials. No kidding it was rejected. Sure - lets just give it to every mutation on the theory the CF science is all the same for every mutation. Very junior move.
Sentiment: Strong Sell
With today's announced rejection by the FDA of the sNDA submitted by VRTX to treat CF patients ages 2-5 with residual function mutations with Ivacaftor, it is clear why it drugs like Ivacaftor cost so much. Vertex bears the financial burden of additional clinical trials to prove what is already apparent with regard to Ivacaftor's efficacy and safety in this CF subpopulation, which is then factored into the drugs pricing, and the affected CF patients are forced to wait even longer to receive this life saving treatment. Interesting that Elijah Cummings and Hilary do not seem to make comments about the impact that these kind of FDA decisions have on drug prices
Good info. VRTX won't say this level. This drug will generate billions for sure and profit margin is very high as well.
Given new break-though treatment, politician will not come on way. They would look coming in way for Innovation. End of day, this is good stock to own.
Sentiment: Strong Buy