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News and Press Releases
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
Research | CFTR Modulators | Drug Pipeline CF Foundation Celebrates FDA Approval of Triple Combination

The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.

| 3 min read
Research | CFTR Modulators | Drug Pipeline FDA Expands Use of Orkambi® to Children With CF Ages 2 to 5

The U.S. Food and Drug Administration approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the F508del mutation.

| 3 min read
CFTR Modulators Positive Early Study Results for Next-Generation CFTR Modulators

New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies. 

| 6 min read