Treated with Kalydeco®. And then?

Question

Hello,
patients receiving a treatment like Kalydeco®, do they have a different life?
I mean by that, do they still need to take many drugs such as Creon®, do they still do a lot of physiotherapy? Or it is case by case?
Is there an increase in life expectancy, but is their life a little more pleasant? Can we hope to have a change in life for our adolescent, if one day searchers find a molecule for the rare mutations?
Thank for your reply. Best regards.
Mum of a 14 year-old adolescent.

Answer

Hello

to remind you, KalydecoTM is available only for 2 years for patients with the G551D mutation (a “gating” mutation, type III). First, this period is very short for saying that some effects exist and are durable, and secondly, we can’t extrapolate these results to other mutations.
On the other hand, we know, each person, each organism reacts differently to a drug even for one type of mutation. The response to a treatment can be modified by external factors.
Yes, the first results of a cohort of patients treated (in the real life) by KalydecoTM show that some are well (regarding the increase of FEV1 and gain of weight…..) and can reduce their others treatments (number of physiotherapy sessions or antibiotics or CreonTM ‘s dose).
I say well "some of them”, as you say it’s case by case, even if the mean follows the good way. But we cannot say, today, if and in what way, KalydecoTM (or similar drugs) will have a long term action and for which mutation.
But there is hope, things move on in research.
- Recently, we know that FDA and EMA have given an approval for a treatment with Kalydeco TM for patients with this 8 following“gating” mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P et G1349D.
- Positive results regarding two phase 3 clinical trials performed with patients over 12 years and homozygous for the deltaF508 mutation, have just been published. During these trials patients received two drugs: a potentiator (KalydecoTM) and a corrector (lumacaftor). Vertex company plans to submit a New Drug Application (NDA) in the U.S., and Marketing Authorisation Application (MAA) in Europe.

Best regards

Virginie Colomb-Jung, M.D
Head of medical department
Vaincre la mucoviscidose

15.09.2014