Kalydeco New Drug Application submited to FDA for R117H for 18 yo and older


New member
It is official the application has been submitted to the FDA for approval. I was told this process takes 6-9 months. Wish this was done 6-9 months ago when the test was completed, but they had to rethink the application since the test endpoints were poorly defined.

I am very thankful that it was not swept under the table after Vertex got good results for the DDF508 combo. To me this is an indicator that Vertex is a responsible company. It is going to be a long 6-9 months, but we have been waiting 17 years, when it was said a cure might be found in 5 years.

Kalydeco is a drug that corrects the underlying cause of CF at the cellular level. This is the first drug that has been able to correct the cellular behavior. Other drugs are simply trying to address the symptoms and not the cause. Since many (apparently most) patients with one R117H do not show symptoms until after 18 the effectiveness was not apparent for the younger age group.