FDA
28th October 2014

FDA Panel Votes in Favor of Expanded Use of Kalydeco (ivacaftor)

On Tuesday October 21st, the Pulmonary Allergy Drugs Advisory Committee (PADAC) 13-2 in favor of expanding use of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug.

FDA Panel Votes in Favor of Expanded Use of Kalydeco (ivacaftor)


On Tuesday October 21st, the Pulmonary Allergy Drugs Advisory Committee (PADAC) 13-2 in favor of expanding use of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug, Kalydeco (ivacaftor) to include a wider range of patients with the rare lung disease. The panel recommended that use of Kalydeco should be approved for patients 6 years and older who have a specific mutation of the R117H gene. Those individuals with this kind of mutation are only about 500 people in the United States. The materials for this meeting can be found HERE

Cystic fibrosis is caused by a defective or absent CFTR protein which results from mutations in the CFTR gene. The current approved indication of Kalydeco covers individuals with a different gene mutation that affects approximately 2,600 people in North America, Europe and Australia. Kalydeco was the first drug approved that addresses the underlying cause of cystic fibrosis rather than only the symptomatic manifestations. Kalydeco helps improve breathing by clearing mucus from the airways.

Dr. Jeffrey Chodakewitz, Senior Vice President and Chief Medical Officer at Vertex, noted that, “Today’s recommendation is a positive and important step toward making ivacaftor available for people ages 6 years and older with the R117H mutation.”

The FDA normally follows the recommendations of its advisory panels, but is not obligated to do so. The Agency is expected to make its decision by the end of 2014.

ISS has over a decade of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComs per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact info@innovativescience.net.

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