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.
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
I agree that a take-over is highly unlikely. Guess, all of us don't understand why VRTX has lost one-third of its value in the last few months. There have been no negative setbacks that might explain the drastic loss in value.
why is VRTX participating in such conferences when they have nothing to talk about, but potential drop-out rates for orcambi? Such discussions will not enhance investors trust in the company.
is it really a worthwhile effort to highlight potential problems of the lead product? VRTX ought to focus on the big picture: sales potential iva and orkambi, VX-661, second gen. compounds, non cf pipeline etc.
Right now VRTX keeps providing the impression that it is facing problems with orkambi. The sp reacts accordingly.
VRTX refusal to issue forecasts for sales of Orkambi has contributed significantly to investors uncertainty. Highlighting potential drop-out rates for patients on Orkambi has confused investors, too. Next CC with quarterly sales of Orkambi in April may bring some clarity. Until then I expect more volatility in sp.
q, VRTX pipeline certainly would warrant a higher share price, but fact is, VRTX has lost much more value than its peers. So there must be specific reasons. In my opinion it is the lack of forecast for Orkambi sales. Management indicates that in real life application Orkambi might not be as good as expected. VRTX needs to give reliable forecasts for Orkambi, if they do not provide that guidance the sp will continue to decline.
Orkambi: Investors can't seem to reach a consensus trajectory for the drug's early launch, except that it's been slower than everyone first believed it would be.
The sell side and the buy side already have cut Orkambi sales estimates this year. The worry now is that another downward revision might be necessary if Vertex's first-quarter earnings coming later this month come up short.