Vertex ($VRTX) 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.
The Massachusetts biotech is pushing forward with a late-stage trial of Kalydeco in tandem with VX-661, evaluating the duo in cystic fibrosis patients who are homozygous or heterozygous for the F508del mutation. Four studies are planned, and next month the company will kick off a trial in patients with two copies of the mutation. Three other studies will launch in the second quarter.
The latest plans are part of what could be a very big year for Kalydeco. The cystic fibrosis treatment has so far been limited saleswise--despite a U.S. price of $300,000--by its tiny patient pool. Last January, CEO Jeffrey Leiden told investors the drug had already reached nearly every eligible CF sufferer in the U.S. and Europe from its original patient population.
Kalydeco has enjoyed boosts since then, most recently by nabbing the FDA's thumbs up to treat patients with the R117H mutation--a nod that will secure 500 new U.S. patients. But that pales in comparison to the lift the drug could see come July, when the FDA is scheduled to decide on the company's one-two punch of Kalydeco plus lumacaftor in patients with the F508del mutation. That genetic abnormality affects 28,000 patients worldwide according to ISI Group's Mark Schoenebaum.
The way some industry-watchers see it, the R117H go-ahead was a sign from U.S. regulators. That FDA approval could mean the agency has a "generally positive view of the program," Sanford Bernstein analyst Geoff Porges wrote in October after an advisory committee meeting. That blessing "bodes well for the approval of the much more important combination next year."
- read the release
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