Recognizing a Milestone: Happy Birthday to Our First Cystic Fibrosis Medicine

Jan 31, 2022

Recognizing a Milestone: Happy Birthday to Our First Cystic Fibrosis Medicine

Today marks an important milestone for our very first cystic fibrosis (CF) medicine — it was 10 years ago today that it was approved in the U.S. Today we celebrate the birthday of this medicine, reflect on the past and think about the future.

Taking on the impossible

Back in 2012 when our first medicine for CF was approved, our team had already been working on the CF program for more than 10 years. I’ve been fortunate to be part of this work from the beginning — combining research and technology, like high throughput screening, with a deep understanding of the underlying cause of the disease. When we started this project, we were often met with a healthy amount of skepticism, but we were determined, and we made a commitment to the CF community. It was with the help of this community — those living with CF and their families and caregivers, the doctors, nurses, investigators and advocates, and most importantly, those who raised their hands to take part in our clinical trials — that we were able to do what many believed was impossible.

The road to drug discovery, development and approval is long and not always straightforward. It takes true collaboration, and I’m so honored to work with a team of trailblazers who have brought not just one CF medicine, but now four CF medicines to people with this disease.

Discovering a medicine is like finding a needle in a haystack. It’s a nearly impossible challenge that few accomplish. The field of CF research has completely shifted throughout out my career, and our research played a huge role in that. I don’t take for granted how incredibly lucky I am to be a part of this for so long.

Fred Van Goor, Ph.D.

The foundation of our R&D model

Multiple new drugs are approved by the FDA every year, so what made this milestone unique? Our scientists understood, at a molecular level, how the cystic fibrosis transmembrane conductance regulator (CFTR) protein functions, and leveraged their expertise in biology and medicinal chemistry to discover and develop potential treatments. And for Vertex? It paved the way for subsequent combinations of CF medicines and became a model for how we approach all our R&D programs — serial innovation, learning from both success and failure, and always remembering that patients come first.

 

I can remember every single molecule I’ve made for the last 20+ years. Each one has taught me something. When 99% of what we do is failure, you have to practice learning from that failure. I think a big part of our success in CF is learning from our failures along the way.

 

Sabine Hadida, Ph.D.

Fulfilling our promise

More than two decades ago, when we first started this work, we made a commitment to people everywhere living with CF. While I learned about the cystic fibrosis transmembrane conductance regulator protein from scientists, I really learned about CF from patients and their families. And it’s this community who is our constant source of motivation.

A lot has changed in the 10 years since that first approval milestone, but our commitment to patients has only grown stronger. It’s the guiding light for who we are at Vertex. As I reflect back on the progress we’ve made and the people with CF who have become part of our Vertex family, we’re inspired to keep pushing our science forward. With 20 ongoing clinical trials in CF, we will continue to relentlessly pursue research, because we’re not done.

Getting to 10 years would not have been possible without the partnership, support and dedication of the CF community. Together with this community, our goal is to discover and develop potential treatments, including genetic therapy and mRNA approaches, for everyone living with this devastating disease. To everyone with CF, we dedicate this milestone to you — we will never stop fighting to fulfill our promise to you.