Orkambi effectiveness in terms of corrected protein count?
- Thread starter cmorgan
- Start date
We can't quantify it exactly. In cells, it restored about 20-30% of protein function. It's thought that we need at least 30-40% function restored to really see a big difference, so Orkambi is kind of right on the cusp. And what happens in cells isn't necessarily exactly what happens in people - it's probably better in cells than people. But the good news is that the triple combos being developed right now restored 70-80% of protein function in cells, so they will be much more effective in people (and are, based on the clinical trial results that have come out so far!)
Wow. Is that 70-80% drug coming out through Vertex or someone else? I heard that Abbvie was working on something in Europe along the same lines as Orkambi.....is that what you're talking about?
Nope it's Vertex! This link has most of the relevant information: http://investors.vrtx.com/releasedetail.cfm?ReleaseID=1033559 as far as how they're doing in patients, and then if you dig around Vertex's site you can find old investor presentations where they show cell data. But the important thing is really this patient data - for people with one or two copies of the dF508 mutation, there was about a 10% increase in FEV1 after just 2 weeks on these combos, which looks very similar to Kalydeco for G551D! I'm happy to go into more detail or answer questions if you have them 