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Twitter Service Announcement: I'll be tweeting pharma updates on #CysticFibrosis for next ~hour while $VRTX holds earnings conference. #VRTX
From press announcement so far, two new points: 1) sNDA for Symdeko for 6-11 homozygotes submitted to FDA late 2018; 2) $VRTX received voucher to get priority review in future. Wondering how they will use this? To get triple first in line? 1/
2/ From Q&A: Keep finding better and better molecules--lots out there to find a safe and amazing triple that benefits both homozygotes and het-mins.
3/ 1st quarter 445 top-line data expected. Looking at "complete profile," which goes to pre-clinical, pk, tolerability, drug-to-drug, as well as efficacy. Pick best to move forward in 1st quarter and mid-year NDA filing. Whether 659 or 445 is selected, data will bein 1st
4/ quarter. VERY SHORTLY 445 data released equivalent with 659, so on track for mid-year NDA. Also, great news for those in socialized countries who often lag 1 - 2 years on access to drugs: Vertex is succeeding in portfolio reimbursement agreements, which means that as soon
5/ EU/UK (Canada, etc.) approve, the patients would have access to drugs--as they do in America. Also, Vertex is working on an opioid alternative, VX150, has had 3-3 successes, doing dose ranging for regulatory reasons to find the lowest efficacious dose. So now figuring out
6/ best dosing. This is in Phase 2B which is great news for those with chronic/acute pain especially given the opioid crisis--so good news for those folks too. (An aside: I really like the women presenter--she's no nonsense and clear and a great addition to presenters).
7/7 That's it from the patient perspective. Now to wait for expansion of Symdeko & release "soon" of second triple results. My go-to on pharma fyi: @matthewherper @megtirrell and @adamfeuerstein END
