CF facts and figures

Cystic fibrosis (CF) is a rare, life-shortening genetic disease. The treatment of CF has changed听dramatically with the advent of cystic fibrosis transmembrane conductance听regulator (CFTR) modulator therapies that address the underlying cause of the听disease.

>20 years

At Vertex, we have worked for more than 20 years to design, discover and develop medicines to treat the underlying cause of CF. 听

>92,000

It is estimated that over 92,000 people have been diagnosed with CF across the U.S., Europe, Australia and Canada, which based on the genetics of the disease represents a significant majority of the global CF population.

~13,000

Beyond the U.S., Europe, Australia and Canada, there are an estimated additional听13,000 people diagnosed with CF, primarily residing in the Middle East and Latin听America.

~90%

Today, our medicines are suitable for approximately 90% of people living with CF.听

>65,000

Through formal reimbursement agreements and other forms of access, such as named patient sales, Vertex medicines are currently treating more than 65,000 patients, representing approximately 2/3 of people diagnosed with CF who are eligible for a CFTR modulator therapy.

99%

In the U.S., our CF medicines are broadly reimbursed through insurance, with more than 99% of eligible patients having access through public and private insurance.

>60

Outside of the U.S., our CF medicines are accessible in more than 60 countries across six continents.

Countries with access to date as of June 2024: Andorra, Australia, Austria, Bahrain, Belgium, Bosnia and Herzegovina, Brazil, Bulgaria, Canada, Chile,鈥疌olombia, Croatia, Cyprus, Czech Republic, Denmark, England and British overseas territories, Estonia, Faroe Islands, Finland, France and French overseas territories, Germany, Greece, Greenland, Hungary, Iceland, Ireland, Israel, Italy, Kuwait, Latvia, Liechtenstein, Luxembourg, Malta, Monaco, Montenegro, Netherlands, New Zealand, North Macedonia, Northern Ireland, Norway, Oman, Poland, Portugal, Qatar, Romania, Russia, Saudi Arabia, San Marino, Scotland, Serbia,鈥疭lovakia, Slovenia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, United States, Uruguay, Vatican City, Wales.*

We are committed to making our CF medicines accessible to patients around the world who can benefit from them, by expanding access to our medicines by geography, by age, and by genotype.听

>45

We have formal reimbursement agreements for our newest triple combination therapy in more than 45 countries around the world, including those with the highest unmet medical need.听

>150

In 2022 and 2023 alone, we signed more than 150 reimbursement agreements, most of them faster than the industry average in the respective country. In many cases, we have portfolio agreements covering future medicines and indications yet to be approved.听

>6,500

More than 6,500 people with CF have been given access to Vertex CFTR modulators at one time free of charge as part of compassionate use/managed access programs worldwide.听

12

We鈥檝e made听our听donated medicines听available at no cost to听patients in听lower income countries on four听continents where access challenges are significant.听Currently the pilot donation program includes 12 countries, and听we are in the process of expanding to additional countries.听We believe that donating medicines with the support of an experienced partner听is the fastest, safest and most reliable way to provide our innovative CF therapies to people with CF in these countries.听Our pilot program was designed to ensure that our medicines are prescribed and utilized appropriately to ensure patient safety. This includes proper diagnosis, ongoing treatment, monitoring and continued pharmacovigilance.

Eligible countries currently included in donation program as of Feb. 2024: Egypt, El Salvador, Honduras, Ivory Coast, Kenya,听Lebanon, Nepal, Sri Lanka, Tanzania, Tunisia, Uganda and Ukraine.鈥

3/5

Three out of every five Vertex employees work in research and development, and we dedicate a majority of our overall operating expenses to R&D to find better medicines and new approaches that could help those not eligible for current therapies.听听


*Disclaimer: Not all therapies and all indications are reimbursed in all listed countries. In some countries, access is granted without regulatory approval in place. This list does not include the countries where Vertex is carrying out pilot donation programs.听

These data points reflect facts as of May 2024.