Vertex Announces FDA Approvals for Expanded Use of Cystic Fibrosis Medicines 

December 31, 2020

Vertex has announced FDA approval of eligibility for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the CFTR gene. SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. This means that more than 600 people with certain rare CF mutations are now eligible to receive these medicines.

“The approval for expanded use of three of our CF medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We remain as committed today to reaching every patient who might benefit from our medicines as when we first started out on this journey 20 years ago…”

“This news underscores Vertex’s investment in innovation to bring transformative medicines to people with serious disease, including cystic fibrosis,” said Kelvyn Cullimore, president and CEO of BioUtah. 

CF is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. It’s a progressive disease, damaging the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. The median age of death is in the early 30s.

BioUtah is working with state leaders to establish a Utah Rare Disease Advisory Council to support patients with rare diseases and help promote research and drug discovery efforts that will lead to life-changing treatments.

In related news, Vertex announced December 28 that its new drug submission for TRIKAFTA® has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines, such as APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.