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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 Enterprise Therapeutics Awarded Up to $7M to Develop Mucus-Clearance Drug for People With CF

The Cystic Fibrosis Foundation has awarded up to $7 million to Enterprise Therapeutics to develop a compound that targets a non-CFTR chloride channel in lung cells. If successful, the drug could help mucus become more hydrated and easier to clear from the lungs of all people with CF, regardless of their CFTR mutations.

| 2 min read
Research Foundation Funds Early Stage Clinical Trial of a Potential Nonsense Mutation Treatment

The Cystic Fibrosis Foundation awarded up to $1.61 million to Eloxx Pharmaceuticals Inc. to conduct a U.S.-based Phase 2 clinical trial of an investigational drug that could potentially treat people with cystic fibrosis who have a nonsense mutation.

| 2 min read
Research | Drug Pipeline FDA Approves Symdeko® for Children Ages 6 to 11

The U.S. Food and Drug Administration has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 with specific mutations.

| 2 min read
Research ICER Announces Assessment of Triple-Combination Therapy

The Institute for Clinical and Economic Review (ICER) is developing an assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The first opportunity to comment runs through Sept. 25.

| 2 min read
About the CF Foundation | Research The CF Foundation Awards Arcturus Therapeutics Up to $15M for RNA Therapy

The Cystic Fibrosis Foundation has increased its award to Arcturus Therapeutics to up to $15 million to develop a mutation-agnostic therapy for people with cystic fibrosis.

| 2 min read
Research | Drug Pipeline Application for Triple-Combination Therapy Submitted to the FDA

Vertex Pharmaceuticals Inc. announced today that it submitted a New Drug Application to the U.S. Food and Drug Administration for the approval of the first triple-combination therapy for cystic fibrosis.

| 1 min read
Research | Drug Pipeline Vertex Selects Triple Combination With VX-445 to Submit for FDA Approval

Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).

| 3 min read
Research | Drug Pipeline FDA Approves Kalydeco for Infants With CF

The U.S. Food and Drug Administration has approved the use of ivacaftor (Kalydeco®) for children as young as 6 months.

| 2 min read