INSM expects to launch ARIKACE on their own in the EU and US upon
approval, NDA submissions are expected for the EU and US in late 2013/early
2014. The company has begun to advance manufacturing and put in place key
members of a commercial team to support the launch of ARIKACE in 2015.
Despite recent approvals of disease-modifying drugs for CF, new antibiotics for CF
associated chronic lung infections continue to represent an unmet medical need.
• We estimate that ARIKACE represents at least a $300-million worldwide
opportunity for the management of chronic Pa infections in patients with CF.
We note there is additional upside to our market estimates should ARIKACE
pricing come in higher than our estimated $6000/cycle estimate. We believe that
the potential for once-daily dosing, effective biofilm penetration and resultant
efficacy could, in our opinion, make ARIKACE the market-leading inhaled antibiotic
in the CF and NTM settings.
Pardon me if this isn't relevant, but has there been any discussion about qualifying for Accelerated Approval for an unmet need, according to the new rules adopted by the FDA for getting drugs to market faster?
From the conference call Q&A:
Joseph Schwartz - Leerink Swann
And also CLEAR-108, is there any way that you could apply those two studies towards a regulatory pathway to be determined in the US?
Sure, so I think the best way to think about the regulatory picture is that we are going to take the 108 data certainly and share it with the FDA. As I’ve said in the past, I think the important things to keep an eye on within the 108 data will be how that data reports out. Do we – my expectation is that we’ll beat the – we will have – meet the end point of non-inferiority.
And my hope is that we will also see some signals within the data that show potentially numerical superiority on key metrics. One of which will be FEV-1 percentage, but the other which will be time to first point of exacerbation, CFU reduction. Those kinds of things, I think show consistent performance and also make an argument of strength and support of any compound.
But to be crystal clear, I don’t have any data. I’m completely blinded at this moment. I just want to highlight what I think is a fair way to review the dataset when it does come out. And when we have that, the strength of that will, I think, be important for the FDA’s receptivity to the use of the CF study in Europe and Canada in their own regulatory considerations and what is necessary for approval.
Our first effort in the US will likely be around NTM. I think the study that we are doing right now, that’s a Phase 2 study, which we’ll report out by the end of the year, which will be supplemented by this 25-patient open label study that we’ve talked about will enable us to go to the FDA and have a dialog about appropriateness of registration and the label. We are hopeful that we’ll be able to do that.
Some of the signals, I think, that you can look for over the year are whether or not we are able to procure two QIDP designation, whether or not we secure a fast track status. We’ve already secured orphan status.