NOTE "experienced experts in PBA from Europe" were pivatal in getting Nuedexta
recommended for approval by the CHMP:
Ritu Baral - Canaccord
Hi guys, thanks for taking the follow-up, just one left quick question about when you guys go in front of CHMP, will your presentation be allowed to include data beyond the STAR trial? Will you be able to bring PRISM registry and clinical data from NUEDEXTA in other – not other indications but PBA secondary to other conditions other than MS, LS? Will you be able to bring that to the table?
Yes, as you know this is an iterative process as opposed to the NDA, right, so we’ve gone back and forth of multiple questions and answers over the past year. And they have seen pretty much all of the data that we know of any including post marketing data in the U.S. from a clinicians prescribing from case [ph] series, from other surveys of commission, the PRISM registry and so forth. And we will remind them of these data. In addition to that, we have contacted and have very important and experienced experts in PBA from Europe in addition to those in the U.S. who are joining us with the meeting. All of them are enthusiastic and then from the European standpoint would love to have a drug like NUEDEXTA to use in their patient safety PBA as it is right now, so they are very keen on seeing NUEDEXTA moving forward.
FACT: it was collaborative effort on the part of countless professional from both Europe and the United States!
Just ask the management team at Vivus and Arena Pharma how easy it is to get a drug approved by the CHMP! If you did not already know, Vivus and Arena have an FDA approved diet drug, but the CHMP voted against that same diet drug coming into Europe due to SAFETY concerns!
"NOTE "experienced experts in PBA from Europe" were pivatal in getting Nuedexta
recommended for approval by the CHMP"
No, we don't know what, if anything, was "pivotal"
If trying to figure out what was weighed more heavily (not very important to do) remember that European PBA experts probably have very little, if any, experience using dex/quin for PBA, unless their centers were involved in the studies 5-11 years ago. And even if they were involved in one of the Phase 3 studies, their whole center would have treated maybe only five patients and none of them would have had non-ALS or non-MS as the underlying neurologic condition. So European PBA experts were probably of little help to Avanir's case.
U.S. PBA experts probably have had more experience treating non-ALS, non-MS PBA but non-study, anecdotal stories have very little weight with pharmaceutical regulatory bodies like the FDA and CHMP so they were probably not "pivotal" either.
CHMP probably made their decision very largely based on the accumulated study data from the double-blind, prospective, multi-center, randomized, placebo-controlled studies and mostly arbitrarily decided to approve the drug for all PBA.
Using 30 mg of dex instead of 20 mg of dex does not add much danger to dex/quin, so I think they were right to approve both doses.
I think the fact that the two dosages were approved merits more discussion, Ray.
I was pleasantly surprised and frankly rather impressed by CHMP's decision. Rather than simply rubber-stamping what the FDA had done (or, worse, approve applications limited to ALS and MS), they appear to have taken advantage of the most recent data available in broadening the label. The application to the EU has therefore actually significantly advanced the franchise of dex + quinidine.