Compassionate use - VX809

Question

Hello,
I turn to you with a question concerning VX-809.
Vertex reported at the presentation of data (Phase 3 Ivacaftor / Lumacaftor from June 24th, 2014) about a "compassionate use" program [comment of the translator: a compassionate use program describes the tolerated usage of a drug that is not licensed for humanitarian reasons]. These do indeed start, when the request for licensing is sent to the FDA or EMA. This should be done in a timely manner (3/4 of this year). Do you have any more precise information about this?
How can one take part in such a program? To whom should one address to? In my center they say, that we as patients will get the drug after market authorization.
To myself: female, 28, vems 35% (after i.v. every 3 months).

Answer

Dear questioner,
we habe contacted the company Vetex concerning the question "compassionate use".
There has been not made a decision yet by Vertex and the FDA (Food and Drug Administration, official agency for food supervision and drug licensing of the United States) if such a program will be realized.
The approach from the side of the patient should be like this, that he informs his CF physician/ CF Center that he would like to take part in such a program, if it would be initiated.
The physician/ center should turn to the Medical Liaison Manager in Germany with the lung function values of his patient (contact can be made from the CF physician via the field manager of the company Vertex).
The data are then going to be reported to Vertex with the information from which center they are coming, so that the CF center can be informed immediately, as soon as there is the possibility for treatment.

Yours sincerely,

Dr. med. Christina Smaczny

13.10.2014