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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.

| 2 min read
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
Research | Drug Pipeline | Advocacy CF Foundation Opposes Right to Try Legislation

The Cystic Fibrosis Foundation recently signed on to a letter with more than 70 organizations in opposition to the Right to Try Act, which passed in the House of Representatives earlier this month.

| 3 min read
Research | Drug Pipeline Positive Early Stage Study Results Announced for Next-Generation Modulator

Phase 2 clinical trial results were announced today for a potential next-generation modulator to treat people with two copies of the most common cystic fibrosis mutation, F508del.

| 2 min read
Research | Drug Pipeline FDA Approves New CFTR Modulator Treatment for Cystic Fibrosis

Tezacaftor/ivacaftor (Symdeko™) is approved for individuals with two copies of the most common cystic fibrosis mutation, F508del, as well as for individuals who have a single copy of one of 26 specified mutations -- regardless of their other mutation.

| 4 min read
Research | Drug Pipeline Next Stage of Triple-Combination CFTR Modulator Development Announced

Today Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.

| 4 min read