Cystic Fibrosis

Question

What are the new therapeutic perspectives?

Answer

Hello

The new therapeutic perspectives concern mostly the therapy of protein, ie re-establish the function of the mutated CFTR protein which is absent or nonfunctional due to the mutation.

Perspective very close, Kalydeco® which is available since 2 years for G551D mutation patients (a “gating” mutation, type III), will soon be prescribed to patients with others “gating” mutation because the European Medecine Agency has given an approval for the treatment by Kalydeco® for patients older than 5 years with one of the following 8 mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P et G1349D.
Positive results regarding two phase3 clinical trials performed with patients over 12 years and homozygous for the deltaF508 mutation, have been published last year (jun). During these trials patients received two drugs: a potentiator (KalydecoTM) and a corrector (lumacaftor). Vertex compagny submitted a New Drug Application (NDA) in the U.S., and Marketing Authorisation Application (MAA) in Europe.

Regarding the gene therapy (replacement of the defective gene with a normal gene) the results of a phase III trial, performed in UK, are strongly expected.

Distant perspectives, a clinical trial regarding patients with at least one non-sense mutation have just begin in France to assess the efficacy of a molecule named Ataluren. Ataluren could correct the mutation and lead to the generation of a normal CFTR protein.
Other studies, planned by Vertex society, with patients F508del heterozygous, are coming.
Novartis society runs (in France and in other country) a clinical trial with a potentiator of CFTR.
Public institutions are also involved in clinical research: a clinical trial with Roscovitine (CFTR potentiator) should begin in France before the end of 2015.

Other french teams (clinicians, but also researchers) work on therapeutic perspectives, pre-clinic results (quite promising) should be confirmed in clinical aspect.

Best regards,
Anna Ronayette
Head of clinical research programs of Vaincre la mucoviscidose

13.03.2015