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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