[10] On 23 January 2019, Ian Smith, the COO and interim CFO of Vertex, was terminated from his position for undisclosed personal behavior that violated established company code of conduct rules.
[20] In April 2024, it was announced Vertex had agreed to acquire the Seattle-headquartered clinical stage biopharmaceutical company Alpine Immune Sciences for $4.9 billion.
On 31 January 2012, Vertex gained FDA approval[22] of the first drug, Kalydeco,[23] to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation.
[24][25] Vertex also studied ivacaftor in combination with another drug (lumacaftor[26]) for the most common mutation in cystic fibrosis (CF), known as F508del, and published the first set of results in 2012.
[34] On 22 October 2019, two months after a new drug application was filed with it, the FDA approved Vertex's Triple-combo therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) for patients 12 and older with at least one F508del mutation.
[51][52][53][54][55] In March 2019, Vertex was legally required to destroy 7,880 packs of Orkambi that reached their expiry dates during price negotiations with the NHS.
[56][57] On October 24, 2019, NHS England agreed to fund wider access to all of the pharmaceutical company's cystic fibrosis medications that were already licensed at that time, such as Orkambi, Symkevi and Kalydeco, and any future indications of these medicines.
[59] On June 30, 2020, Vertex and NHS expanded the agreement to include reimbursement of Kaftrio (the name used for marketing Trikafta in the UK), making England one of the first countries in Europe to fund it.
[60] In October 2023, NICE declined to recommend Vertex's cystic fibrosis drugs Kaftrio, Symkevi, and Orkambi for use in CF patients, citing their high costs as beyond the acceptable range for NHS resources.
About 27% (51,322) of the estimated CF patients were treated with Trikafta, of whom nearly half (49%) lived in the U.S., 42% in Europe, and 9% in Canada, Israel, Australia, and New Zealand combined.
Price negotiations and managed entry agreements (which for example link reimbursement to clinical performance) are common strategies used by countries to make Trikafta more affordable, though the details of these arrangements are often confidential.
[69] In the late 1990s the Bethesda-based Cystic Fibrosis Foundation, encouraged by then-President Robert Beall, began investing in Vertex— when it was a small start-up biotechnology company— to help fund the development of Kalydeco in the form of venture philanthropy.
[73][74] Proponents of venture philanthropy say the high financial return helps speed drug development and also provides potential monetary rewards that can go to more research.
"[76] Twenty-nine physicians and scientists working with people with cystic fibrosis (CF) wrote to Jeff Leiden, CEO of Vertex Pharmaceuticals to plead for lower prices.
[77] We are aware of the financial complexities of the huge expenses for R & D with respect to the small number of patients or the market system that enables these advances to become reality.
This action could appear to be leveraging pain and suffering into huge financial gain for speculators, some of whom were your top executives who reportedly made millions of dollars in a single day (Boston Globe, 29 May).The company responded in an email that "while publicly funded academic research provided important early understanding of the cause of cystic fibrosis, it took Vertex scientists 14 years of their own research, funded mostly by the company, before the drug won approval.