The U.S. Food and Drug Administration approved Kalydeco® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.
- About the CF Foundation
- Public Policy
- Drug Pipeline
- Get Involved
- Genetic Therapies
- Our Research Approach
- Clinical Trials
- Health Care Reform
- North American CF Conference
- Care Centers
- Our Advocacy Work
- Patient Assistance
- Cystic Fibrosis Foundation Therapeutics (CFFT)
- Patient Registry
- Infection Prevention and Control
- Lung Transplantation
- In the Spotlight
- Coronavirus (COVID-19)
- Diversity, Equity, and Inclusion
- Intro to CF
- Clinician Resources
- Great Strides
- In Memoriam
- Adult Care
- Care Team
- CF-Related Complications
- CF Related Health Conditions
- Fertility and Reproductive Health
- Researcher Resources
- (-) CFTR Modulators
With this approval, approximately 2,250 children in the U.S. will be eligible for Trikafta® (elexacaftor/tezacaftor/ivacaftor), including more than 900 who will have access to a CFTR modulator for the first time. The Cystic Fibrosis Foundation believes it is beneficial to start people with cystic fibrosis on modulators as early as possible to help prevent lung damage and the onset of complications.
Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.
The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
The CF Foundation has successfully completed a sale of its remaining stake in royalties related to Vertex's CFTR modulators for an upfront payment of $575 million and a potential future payment of $75 million, bringing additional resources to the fight against CF.
Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
On Aug. 27, leaders from the Cystic Fibrosis Foundation, along with several people from the CF community, participated in a virtual public meeting hosted by the Institute for Clinical and Economic Review to provide insights and expertise on the value of CFTR modulators.
The Institute for Clinical and Economic Review (ICER) recently posted a draft evidence report to outline their initial assessment of the clinical effectiveness and value of CFTR modulators, including elexacaftor/tezacaftor/ivacaftor (TrikaftaTM). The document is open for public comment through March 18.
The Cystic Fibrosis Foundation announced today that it has licensed a compound to the biopharmaceutical company AbbVie to develop into a potential CFTR modulator treatment.