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News and Press Releases
CFTR Modulators FDA Approves Kalydeco for Infants as Young as 1 Month

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.

| 4 min read
CFTR Modulators FDA Approves Trikafta for Children Ages 2 Through 5 Years With Certain CF Mutations

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.

| 3 min read
CFTR Modulators FDA Approves Orkambi for Children With CF Ages 1 to 2 Years

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.

| 3 min read
CFTR Modulators FDA Approves Trikafta for Children Ages 6 Through 11 With Certain 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.

| 4 min read
Research | CFTR Modulators FDA Approves Expansion of Modulators for People With Certain Rare Mutations

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.

| 3 min read
CFTR Modulators | About the CF Foundation A Message From Our CEO

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.

| 3 min read
Research | CFTR Modulators Roche Acquires Potential CF Therapies; Plans to Develop Alternatives to CFTR Modulators

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.

| 3 min read
CFTR Modulators | About the CF Foundation | Clinician Resources CF Foundation Leaders Provide Expertise During ICER Public Meeting

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.

| 3 min read
CFTR Modulators ICER Issues Draft Evidence Report for Assessment of Trikafta

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.

 

| 2 min read
Research | CFTR Modulators Cystic Fibrosis Foundation Is Exploring New CFTR Modulators

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.

| 2 min read