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
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.
based on the price for the royalties that the cf foundation received a price target of $180 seems reasonable. However, these days the sp seems to be not based on fundamentals but on algorithms that hedge funds use. let's hope that the market some day will again value fundamentals.
wow, that's not bad: In preclinical studies, the combination showed up to a six-fold greater increase in chloride transport in human bronchial epithelial (HBE) cells than Vertex Pharmaceuticals' ORKAMBI (lumacaftor/ivacaftor). - sixfold better!
VRTX sp performance during the last month compared to other big biotech's indicates indeed that "the market" is expecting major negative information, Clintons initiative certainly can not explain the steep decline in sp, VRTX is not affected by reduction on patent extensions for biologics, let's see what next cc will bring
don't blame the politicians, nobody can justify what Turing did, raise the price of an old drug from $13.5 to $750!
without info on the non-cf pipeline it is not possible to give a substantiated guess on a buy-out offer. My wild guess is somewhere between $170 and $200 - if an offer will be made, which I doubt.
J. Leiden: " The residual function arm always had in it both a placebo and ivacaftor monotherapy compared with 661 plus ivacaftor."
I was not aware of iva mono versus iva+661, to me it seems that FDA asked for that arm, given their doubt on Orkambi
Q, there is no doubt that Kalydeco and Orkambi are major achievements in treatment of CF that are a blessing for many patients. But that doesn't relief management from shaping the future of the company and to create a sustainable stream of revenue. In my opinion, VRTX has not offered much information others than referring to announcements to be made in the coming years. Second generation correctors were supposed to enter clinic in 2014. Where are they? Galapagos has a better version of Kalydeco in the clinic. Where is VRTX back-up for Kalydeco? Where are clinical candidates outside CF? Why go after cancer candidates that improve chemotherapy when the company's focus is on orphan drugs?
I am a long time investor in VRTX, cost average of my shares is below $20, so I do not complain about my capital gain, but I dare to criticize the management of the company I am invested in when I feel somethings missing.
this has been my strongest concern for quite some time (years!):
wonder when they will bring more compounds to the clinic. The cancer candidates seem to be locked up in phase I and do not really fit Leiden's strategy to focus on orphan drugs.