News

News and Press Releases
Research | Drug Pipeline | Medications FDA Accepts Vertex Application for Expansion of Trikafta to Include Children ages 6-11

Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021. 

| 2 min read
Drug Pipeline FDA Approves Kalydeco for Infants as Young as 4 Months

The U.S. Food and Drug Administration has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.

| 3 min read
Drug Pipeline | Clinical Trials Vertex Announces Positive Study Results for Trikafta in Children Ages 6-11

Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.

| 2 min read
Drug Pipeline Vertex Asks FDA to Expand CFTR Modulators to Additional Mutations

Vertex Pharmaceuticals Inc. has submitted three supplemental New Drug Applications to the U.S. Food and Drug Administration (FDA) to expand eligibility for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) to additional rare CFTR mutations.

| 2 min read
Drug Pipeline Positive Phase 3 Results of Trikafta Provide Further Evidence of Efficacy

Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 study results for Trikafta® (elexacaftor/tezacaftor/ivacaftor) for people with cystic fibrosis ages 12 years and older who have one copy of the F508del mutation and one gating or residual function mutation.

| 2 min read
Drug Pipeline | About the CF Foundation CF Foundation Awards Leading Research Organization Funding to Screen for Potential B. Cepacia Therapy

The Cystic Fibrosis Foundation awarded nearly $700,000 to Calibr, the drug discovery and development arm of Scripps Research, to screen for a compound that could be used to treat Burkholderia cepacia complex infections. 

| 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
Drug Pipeline ICER Posts Draft Scoping Document for Assessment of Triple Combination Therapy

The Institute for Clinical and Economic Review (ICER) recently posted a draft scoping document to outline their planned assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The document is open for public comment through October 21.

| 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 | 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