Vertex ($VRTX) 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.
The agency's panel of experts voted 12 to 1 to recommend greenlighting Orkambi--which combines the CF star with experimental med lumacaftor--in patients ages 12 and older who have two copies of the F508del mutation in the CFTR gene. With that in mind, U.S. regulators will decide by a July 5 deadline whether to give the therapy a go-ahead.
If they do, it'll mean 8,500 potential new patients for the Massachusetts biotech, which has relied on label expansions to keep Kalydeco sales growing. Since hitting the market in January 2012 as a treatment for CF patients with the G551D mutation, Vertex has nabbed indications in patients with the RH117H mutation and 8 others.
|Vertex CCO Stuart Arbuckle|
Even If Orkambi passes muster with the FDA in F08del patients, other challenges may lie ahead. As backlash against pricey meds mounts, Vertex has already had to justify its $300,000-plus Kalydeco price tag. Payer pressure may ramp up if Orkambi--which Vertex thinks can help it reach 10 times as many patients--is priced similarly.
But Vertex has already started prepping for that possibility. As Chief Commercial Officer Stuart Arbuckle told investors in February, the company is scaling up its patient services team and "investing in disease education to help payers estimate the number of eligible patients they may have in their plans."
- read Vertex's release
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