FDA approves Orkambi for children ages 6 through 11...

[Imogene]

[h=1]U.S. Food and Drug Administration Approves ORKAMBI[SUP]®[/SUP] (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation[/h] -Approximately 2,400 children ages 6 through 11 have two copies of the F508del mutation in the U.S.-
- Vertex revises ORKAMBI revenue guidance for 2016 -




http://www.businesswire.com/news/ho...tration-Approves-ORKAMBI®-lumacaftorivacaftor

 

[Ratatosk]

This is exciting news! DS has been on Orkambi since last year and we've seen improvements in growth (height and weight), lung function, normal flora in cultures.

I recently read an article about a child on the west coast who had chronic sinus issues, chronic infections to the extent they were running out of options with surgeries, antibiotics, etc., concerned the infection could get into the brain. She was able to get on Orkambi last fall and hasn't had any hospitalizations and has had clear culture results.

IMO, while not a cure it's another tool in the toolbox to buy time until a long term treatment or cure can be found.

 

[Carriekit]

Hi. Does anyone have an idea of when Orkambi could be approved for those having one DF508 and another mutation in the same class? My daughter has DF508 and 2184insA (same class A mutation as DF508).

 

[Ratatosk]

As far as I know, Orkambi is primarily for ddf508, though I seem to recall a couple people being able to get it off label --- meaning they didn't have two copies. Not sure if they saw any benefits.

The CFF has a list of drugs currently under development, including some focusing on those with one copy delta f508. Here's the link:

https://www.cff.org/trials/pipeline