Vertex ($VRTX) 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.
With Kalydeco's newest label expansion will come 500 potential new patients in the U.S., where the therapy carries a $300,000 price tag. It's a welcome lift for the drug, which as of January had reached nearly every eligible CF sufferer in the U.S. and Europe from its original patient pool, company CEO Jeffrey Leiden told investors at the time.
"Today's approval marks an important milestone for people with the R117H mutation who will now have a medicine to treat the underlying cause of their disease for the first time," Chief Medical Officer Jeffrey Chodakewitz said in a statement. "We are now one step closer to reaching our goal of providing new medicines to many more people living with cystic fibrosis."
The thumbs up from U.S. regulators follows a positive FDA committee recommendation from October, which saw a panel vote 13-2 in favor of approval. And some industry watchers, like Sanford Bernstein analyst Geoff Porges, saw that positive sign as a "soft indicator" for the FDA's sunny views on Vertex' wider program.
With quite a bit riding on its next label expansion, the Massachusetts company certainly hopes that's the case. Vertex is looking to bring a Kalydeco-plus-lumacaftor combo to patients with the F508del mutation--one that affects more than 28,000 patients worldwide, ISI Group's Mark Schoenebaum says.
- read Vertex's release
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