Ivacaftor and Lumacaftor

Question

The results of the phase 3 study have been published a few days ago. There were slight improvements in the 24-weeks study. How can I judge the resutls realistically for the future (timely manner, in case of long-term intake change of the mean life expectancy)?

Many thanks for your time

Answer

Dear questioner,
I assume that you refer to the data that has been published by the company Vertex itself in the internet (investors.vrtx.com/releasedetail.cfm?ReleaseID=856185).

The company is reporting consciously good things about the outcome of the 24-weeks phase 3 study, please notify also, which target group is meant here ("investors"!.vrtx.com).
The so-called "disclaimer" (that you find in case of the darwing of lottery numbers always at the end of the program in the form of the statement "all information is subject to change"), you can find at the end of the page:
investors.vrtx.com/releasedetail.cfm?ReleaseID=856185

" Special Note Regarding Forward-looking Statements ----- This press release contains forward-looking statements [.....] regarding Vertex's plans to submit regulatory applications for the approval of lumacaftor in combination with ivacaftor in multiple countries [.....] in the fourth quarter of 2014 for people ages 12 and older who have two copies of the F508del mutation. While Vertex believes the forward-looking statements contained in this press release are accurate [.....] as of the date of this press release [.....] there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. [.....] Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. "

Short summary:
1. The data is preliminary
2. Insights can be made that are contradicting the data and
3. Vertex is not feeling responsible for correcting the text of this website

Of course it is not possbile for anyone, not even for the company Vertex itself either, to make conclusions about long-term effects on the basis of the 24-weeks study - on the one hand positive effects as well as negative effects can occur only in case of many years of therapy, on the other hand not all drugs are as efficient in all people, so that for the single case a prediction can hardly be made after a testing phase with comparatively few people.

I propose that you turn to a CF physician of one of the study centers here in Germany that have been taken part in the international study fot the combination therapy of Ivacaftor and Lumacaftor, in order to get an immediate and realistic judgement of the new therapeutic options. For this purpose you find the participating centers with the abbrevetions " VX09-809-102", "VX12-809-103", "VX12-809-104", "VX12-809-105" on the following site [German website]: "muko.info/mukoviszidose-institut/klinische-studien/cf-studien-in-deutschland.html"

Best regards,
Frauke Stanke

21.08.2014