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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
Drug Pipeline | Research Foundation Awards Up to $5.1 Million for NTM Drug Discovery and Development

The Cystic Fibrosis Foundation awarded up to $5.1 million to TB Alliance to advance a drug discovery program in partnership with Johns Hopkins University. The effort will identify compounds with the potential to treat nontuberculous mycobacteria infections that are increasingly found among people with CF.

| 3 min read
Research | Drug Pipeline New, Positive Results Announced for Triple-Combination Therapy

Today, Vertex released positive preliminary clinical trial results for the potential triple-combination therapy VX-445 plus tezacaftor/ivacaftor (Symdeko®).

| 3 min read
Drug Pipeline CF Foundation Expands Funding Up to $4.5 Million to Talee Bio Inc. to Develop CFTR Mutation-Agnostic Gene Therapy

The Cystic Fibrosis Foundation awarded Talee Bio Inc. up to $4.5 million to develop methods for delivering potential gene therapy treatments to the lungs. 

| 2 min read
Research | Drug Pipeline | Clinical Trials Positive Late Stage Study Results for Next-Generation, Triple-Combination Modulator

Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.

| 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
Research | Drug Pipeline Drug Shows Promise in Reducing Inflammation in People With Cystic Fibrosis

Celtaxsys Inc. reported promising results today from an early stage clinical trial of a potential anti-inflammatory drug for people with cystic fibrosis.

| 2 min read
Research | Drug Pipeline | Clinical Trials Phase 2 Study Shows Promising Results for Potential Mucus Clearance Drug

A new drug intended to make it easier to clear mucus from the lungs of people with cystic fibrosis showed promising results in a Phase 2 clinical trial. The drug, called SPX-101, blocks the overactive sodium channels that line CF lung cells.

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Research | Drug Pipeline | Advocacy President Trump Signs Right to Try Legislation Into Law

Yesterday, President Donald J. Trump signed the Right to Try bill into law. The House approved the bill last week and the Senate passed it last summer.

| 3 min read