#TBT – Cystic Fibrosis Patient Testimony on Orkambi
Orkambi is a combination therapy for treatment of cystic fibrosis. The FDA approval for the therapy was announced on July 2nd, 2015.
In this StratAcuity: The Science of People video, watch this Orkambi testimony before the Open Public Hearing OPH portion of the May 12, 2015, Pulmonary-Allergy Drugs Advisory Committee (PADAC) meeting in Washington, D.C. by cystic fibrosis patient Kate Marshall.
The 15-year-old has two copies of the D508 gene, which Orkambi, (the combination of Ivacaftor and Lumacaftor) treats.
In March, the U.K. National Institute for Health and Care Excellence (NICE) issued a draft guidance against recommending Orkambi (lumacaftor-ivacaftor) for treating cystic fibrosis (CF) since its cost is too high for the low benefits it brings.
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