|Industry||Pharmaceuticals & Biotherapeutics)|
|Revenue||US$3.04 billion (2018)|
Vertex Pharmaceuticals, Inc. is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in South Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, near Oxford, England.
Since late 2011, Vertex has ranked among the top 15 best performing companies on the Standard & Poor's 500. Vertex shares increased 250 percent in the same period. In January 2014, Vertex completed its move from Cambridge, Massachusetts to Boston, Massachusetts, and took residence in a new, $800 million complex. Located on the South Boston waterfront, it marked the first time in the company's history that all of the roughly 1,200 Vertex employees in the Greater Boston area worked together.
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. In June of the same year, Vertex announced it would acquire Exonics Therapeutics for up to $1 billion and collaborate with CRISPR Therapeutics, boosting its development of treatments for Duchenne muscular dystrophy and Myotonic Dystrophy Type 1.
In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash. Semma Therapeutics created a "small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out."
On 1 April 2020, former Chief Medical Officer, Reshma Kewalramani, became President and Chief Executive Officer of Vertex Pharmaceuticals. Former CEO and President Jeffrey Leiden transitioned to the role of Executive Chairman of the Board of Directors, as of 1 April 2020. Dr. Leiden will serve as Executive Chairman until April 2023.
Ivacaftor, lumacaftor and tezacaftor
In 2012 ivacaftor, was designated as an orphan drug, identifying cystic fibrosis as affecting fewer than 200,000 people in the United States. On 31 January 2012, Vertex gained FDA approval of the first drug, Kalydeco, to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation. In the US, 30,000 people have cystic fibrosis.
Vertex also studied ivacaftor in combination with another drug (lumacaftor) for the most common mutation in cystic fibrosis (CF), known as F508del, and published the first set of results in 2012. Vertex produced the drug after 13 years of research and development, with $70 million in support from the Cystic Fibrosis Foundation.
In the UK, the company provided the drug free for a limited time for certain patients. Subsequently, the hospitals decided to continue to pay for the drug for those patients. UK agencies estimated the cost per quality adjusted life year (QALY) at between £335,000 and £1,274,000—far above the NICE thresholds of £20,000-£30,000.
On 5 November 2014 Vertex announced the submission of a New Drug Application (NDA) to the FDA for a fully co-formulated combination of lumacaftor and ivacaftor for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In 2015, FDA approved the combination of lumacaftor and ivacaftor to treat CF in patients 12 years and older, who have the F508del mutation. The combination drug is marketed under the tradename Orkambi.
On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis. On 12 February 2018, the FDA approved the combination, marketed as Symdeko.
Triple combination treatments
In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor. In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population. On 22 October 2019, the FDA approved Vertex's Triple-combo therapy "TRIKAFTA (elexacaftor/tezacaftor/ivacaftor)" for patients 12 and older with at least one F508del mutation. This approval came only 2 months after the NDA was filed. Trikafta is approved for approximately 90% of patients with cystic fibrosis.
- In 2015, Vertex entered into a research collaboration with CRISPR Therapeutics to develop gene-editing therapies for genetic diseases. In May 2020, CTX001 has received Orphan Drug Designation from the U.S. Food and Drug Administration for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia.
- In 2016, the company entered into a collaboration with Moderna to develop new mRNA-based therapeutics to treat CF; Vertex paid Moderna $20M in cash and provided an additional $20M in cash in exchange for a convertible note that Vertex can cash in for stock. Vertex also promised to pay up to $275M in milestone payments.
- In 2019, Vertex established a research collaboration with Arbor Biotechnologies to discover novel proteins to advance the development of new gene-edition therapies for cystic fibrosis and four other diseases.
In 2014, Vertex discontinued telaprevir. In May 2011, the Food and Drug Administration (FDA) approved the drug telaprevir (Incivek), an oral treatment for hepatitis C marketed by Vertex. Development and commercialization of telaprevir is shared with Johnson & Johnson for European distribution and Mitsubishi for Asia. Telaprevir is a protease inhibitor.
In 2015, several media outlets reported CEO Jeffrey Leiden's 2014 compensation to be approximately US$48.5 million. Vertex shareholders opted for a reduction in CEO compensation in 2015 and 2016, resulting in 2016 earnings of US$17.4 million.
Pricing in the UK
Criticism of Vertex-based around claims that the pricing of their groundbreaking cystic fibrosis drug Orkambi (priced at US$135,000 to US$270,000 per patient per year, depending on the patient's country), limited access to the drug for children and young adults with cystic fibrosis, resulted in campaigns by concerned parent groups and allegations of unfair pricing by the UK's National Health Service.
On October 24, 2019, Vertex reached an agreement with NHS England which provided improved access to all of the pharmaceutical company's cystic fibrosis medications that are already licensed, such as Orkambi, Symkevi and Kalydeco, and any future indications of these medicines. The deal was finalized soon after Vertex signed reimbursement deals elsewhere, including NHS Scotland, Spain and Australia. The NHS in Wales and Northern Ireland will be offered equivalent pricing terms.
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. The total investment amounted to $150 million. In 2014, the CF Foundation sold the rights to the royalties of the drugs for $3.3 billion, twenty times the foundation's 2013 budget. 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.
By 2015 the annual price of Kalydeco had been increased to more than $300,000 per patient.
According to an article published in the Milwaukee Journal Sentinel Vertex executives "grossed more than $100 million by cashing in stocks and stock options" and at "one point, the value of company's stock increased more $6 billion in a single day."
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.
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. Yet – notwithstanding all your patient support programs – it is at best unseemly for Vertex to charge our patients' insurance plans (including strapped state medical assistance plans), $294,000 annually for two pills a day (a 10-fold increase in a typical patient's total drug costs). 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).— David M. Orenstein, MD et al.
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."
On 15 April 2015 in Cambridge, MA, Joan Finnegan Brooks of the Cystic Fibrosis Foundation, spoke about the role of Vertex and venture philanthropy to a panel of biotech leaders hosted by Life Sciences Foundation on the topic of patient advocacy in the biotech industry. While Brooks, who has Cystic Fibrosis, expressed gratitude for Vertex' development of Kalydeco, she observed that "More than 25% of people are saying (in a surveys conducted by the CFF) that they are skipping medications or delaying medications or skipping doctor appointments because of cost of care issues." She added that "one of the things that the Foundation has done," is to "develop resources that can help patients bridge that gap through patient access programs, and so forth."
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The triple therapy, which has just received approval from the FDA in the US, is thought to be effective in up to 90% of CF patients.
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