Kalydeco Receives Recommendation for Approval in the European Union

May 25, 2012

The European Medicines Agency (EMA) announced today that it has recommended the approval of the CF drug Kalydeco™ for people with the G551D mutation ages 6 and older in the European Union.

The recommendation is being sent to the European Commission, which has the authority to approve medicines in the European Union.

The European Commission generally follows the recommendation of the EMA and typically makes a decision on approval of a drug within three to four months.

Kalydeco is the first drug that treats the underlying cause of CF — a defective gene and its protein product, known as CFTR. Earlier this year, the FDA approved Kalydeco for people with the G551D mutation ages 6 and older in the United States. 

The drug was developed by Vertex Pharmaceuticals Inc., with significant scientific, clinical and funding support from the Cystic Fibrosis Foundation.

“The EMA’s recommendation is great news and marks an important step forward in bringing therapies that target the basic defect in CF to more people with the disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Approval of Kalydeco in the European Union will give people with the G551D mutation in those countries the opportunity to experience the same improvements in health seen among those who are now benefiting from the drug in the U.S.”

Additional Resources