Vertex is looking to expand its market for cystic fibrosis drug Kalydeco tenfold, but to do that, it needs the FDA's backing. On Tuesday, it got one step closer, grabbing an advisory committee nod for a new Kalydeco combo.
As an FDA review nears for Vertex's Kalydeco-lumacaftor tandem--approval of which could help Kalydeco reach 10 times the number of cystic fibrosis patients it currently does--the agency's staffers have some concerns.
Sky-high drug prices have been causing their fair share of consternation among payers lately, and industry watchers have suspected for a while now that Vertex will stir the pot when its new cystic fibrosis combo--expected to win FDA approval later this year--hits the market. But just how much will payers need to shell out for the new med over time? Prime Therapeutics has some ideas.
Arkansas Medicaid officials have wrapped up a legal dispute over Vertex's cystic fibrosis drug Kalydeco, settling to resolve claims the state denied patients the treatment based on its high price tag. And while it's good news for the drugmaker, this may be just the first in a series of cost-control battles to follow.
Vertex is on the verge of something very big with lumacaftor, a drug that could revolutionize cystic fibrosis treatment for nearly half the world's patient population. But as it awaits approval and looks ahead to launch, it's taking care to avoid the payer pushback Gilead sparked with its pricey next-gen hep C med Sovaldi.
Vertex is already awaiting the FDA's decision on a Kalydeco combo that could exponentially amp up its cystic fibrosis patient pool. But in the meantime, it announced Sunday evening, it's starting in on a Phase III program for yet another Kalydeco pairing.
Vertex will start the new year with a group of new eligible patients for cystic fibrosis drug Kalydeco, thanks to a Monday green light from the FDA.
Vertex will start the new year with a group of new eligible patients for cystic fibrosis drug Kalydeco, thanks to a Monday green light from the FDA. The agency has OK'd the med in patients with the R117H mutation, a nod analysts say will boost Kalydeco in the present and also bodes well for its future.
Despite falling short of its primary endpoint in a trial to examine its effects in cystic fibrosis patients with the R117H mutation, Vertex's Kalydeco has scored an FDA advisory committee recommendation for approval in that population. And the way some see it, that's a sign of much, much bigger approvals to come.
Vertex' orphan drug Kalydeco boasts a comparatively small patient pool that the drugmaker is trying to swell, with an eye on adding cystic fibrosis patients with the R117H mutation. But first, it'll have to get the FDA's OK, and new briefing documents suggest the agency still has some doubts.