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Compassionate use program for deltaF508 patient
- Question
- Hello
In the press release dated on June 24th, 2014, the Vertex company talked 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] for people who really need a new medicine (I get i.v. antibiotics every 2 months and my FEV1 is actually 30% of the predictive value).
Do you have some news? When could this program start? How to get in?
I’m not expecting a real improvement but a stabilization of my health.
Knowing that submission to the FDA [US Food and Drug Administration] (and EMA [European Medicines agency]?) has the status of fast track designation, when can we expect the agreement of these two regulatory instances?
Thank for your reply.
A CF patient of 32 years.
- Answer
- Hello
At the moment, we haven’t information regarding a "compassionate use program" for patients with the deltaF508 mutation, who need the combination regimen (VX770 + VX809).
Best regards,
Anna Ronayette
Head of clinical research programs of Vaincre la mucoviscidose
- 06.10.2014
