Vertex' ($VRTX) patient pool for cystic fibrosis drug Kalydeco is getting wider--250 patients wider, to be specific. The European Medicines Agency has green-lighted the drug--originally approved for CF patients with at least one copy of the G551D mutation--for 8 additional mutations, spelling new revenue potential for the orphan drugmaker.
The EMA's blessing follows a Phase III study of 39 people with non-G551D gating mutations. Patients with the G178R, S549N, S549R, G551S, G1244E, S1251N and S1255P mutations--those for which Kalydeco won the EMA's favor--showed "rapid, significant and sustained improvements in lung function and other measures of disease," President-Elect of the European Cystic Fibrosis Society Kris De Boeck said in a statement.
Next up: making Kalydeco available to those 250 newly eligible patients as soon as possible, which the company will do by teaming up with national authorities, Vertex SVP Simon Bedson said in a statement.
The new approval will provide a welcome revenue boost in Europe, where Vertex has already reached nearly all eligible patients with the G551D mutation. Europe is home to about 100 more eligible patients than the U.S., where the FDA in February approved the same indication.
Both those markets should help Kalydeco toward the $520 million to $550 million 2014 revenue guidance the company confirmed with its second-quarter earnings announcement earlier this week. Vertex expects Kalydeco to provide the bulk of the top-line haul, forecasting revenues for the drug within the $470 million to $500 million range.
- read the release
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