Letter to the Community on Orkambi Approval

Today we received the exciting news that the U.S. Food and Drug Administration has approved Orkambi for people with cystic fibrosis ages 12 and older who have two copies of the F508del gene mutation, the most common CF mutation.

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We are thrilled with the FDA's decision. The approval marks a major milestone in our decades-long effort to spur discovery and development of innovative therapies that target the root cause of CF to improve key symptoms of the disease.

More than 1,100 people with CF around the world volunteered for the clinical trials that led to Orkambi's approval, and we are deeply grateful for their invaluable contributions. We also thank the incredible team of scientists and clinical researchers and, most of all, the tens of thousands of families, friends, volunteers and donors whose hard work and generosity fueled this long, hard effort.

Orkambi is the second approved drug to emerge from the CF Foundation's research program with Vertex Pharmaceuticals. The first, ivacaftor, is now available as a stand-alone therapy to people who have certain rare CF mutations. With Orkambi's approval, more than 10,000 people living with CF can benefit from a drug that treats the underlying genetic cause of their disease.

Today's announcement gives us much to be proud of, along with tremendous momentum as we move forward urgently in our efforts to find powerful new therapies for all people with CF.

As we celebrate this exciting news, I hope you also feel proud of the vital role you have played in this major step forward. With the passionate commitment and support of this extraordinary community, I am confident that we will continue to forge new paths and reach our ultimate goal: a cure for all people with CF.

Sincerely,

Robert J. Beall, Ph.D.

President and CEO

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Topics
About the CF Foundation | CFTR Modulators | Genetic Therapies
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