|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 January 23, 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.
On July 25, 2019 Vertex Pharmaceuticals announced that Jeffrey Leiden will transition to the role of Executive Chairman of the Board of Directors, effective April 1, 2020. At that time, current Chief Medical Officer Reshma Kewalramani will succeed him as President and Chief Executive Officer. Dr. Leiden will serve as Executive Chairman until April 2023. 
Notable pharmaceuticals and treatments
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.
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 January 31, 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 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 November 5, 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 Orkambi lumacaftor/ivacaftor to treat cystic fibrosis (CF) in patients 12 years and older, who have the F508del mutation.
On March 28, 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis. On February 12, 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.
In 2015, Vertex entered into a research collaboration with CRISPR Therapeutics to develop gene-editing therapies for genetic diseases. 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 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 (costing in the region of US$135,000 to US$270,000 per patient per year, depending on the patient's country), limits 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 .
The NHS offered Vertex approximately £500 million (US$655 million) over five years and £1 billion over ten years, in mid-2018, the largest financial commitment to a pharmaceutical firm in NHS history, for access to Vertex drugs including Kalydeco, Orkambi, tezacaftor/ivacaftor and future medicines. Vertex turned down the proposal saying it was determined to find a solution "that allows the NHS to provide patient access...and also allows Vertex to continue its research and focus on a cure for CF and other serious diseases." Non-disclosure agreements prevent the publication of the Vertex proposed pricing model to the public. Leiden wrote to the UK Prime Minister Theresa May, suggesting he might consider reducing investment by Vertex in the UK as a result of the offer.
Kalydeco pricing has also faced criticism.
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 cost of Kalydeco had risen 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, May 29).— 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 April 15, 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." 
- "Vertex Pharmaceuticals Incorporated". Hoover's. Retrieved 24 June 2012.
- Weisman, Robert (24 April 2011). "Rearranging the Corporate DNA". Boston Sunday Globe. p. G1. Retrieved 25 June 2012.
- Werth, Barry (1994). The Billion-Dollar Molecule: One Company's Quest for the Perfect Drug. New York: Simon & Schuster. ISBN 0671510576. OCLC 32047662. The Billion-Dollar Molecule at Google Books.
- Werth, Barry (2014). The Antidote: Inside the World of New Pharma. New York: Simon & Schuster. ISBN 9781451655667. OCLC 859375019.
- "Vertex Pharmaceuticals Reports Fourth Quarter and Full Year 2004 Financial Results and Full Year 2005 Guidance". Vertex Pharmaceuticals. Retrieved 30 May 2019.
- "Vertex Pharmaceuticals on the Forbes Global 2000 List". Forbes. Retrieved 30 May 2019.
- Robert Weisman (17 April 2015), Vertex chief's compensation totaled $36.6 million last year: Total included a retention bonus, Boston Globe, retrieved 19 July 2015
- Staff, Robert Weisman Globe; February 4; 2014; Comments, 12:00 a m Email to a Friend Share on Facebook Share on TwitterPrint this Article View. "New complex unites Vertex employees - The Boston Globe". BostonGlobe.com. Retrieved 30 May 2019.
- Owens, Jeremy C. (23 January 2019). "Vertex terminates COO for 'personal behavior'". MarketWatch. Retrieved 25 January 2019.
- "Vertex's Reshma Kewalramani takes CEO post as longtime vet Jeff Leiden moves to new role". Endpoints News. Retrieved 31 July 2019.
- "Vertex Pharmaceuticals' Jeffrey Leiden to step aside as CEO". STAT. 25 July 2019. Retrieved 31 July 2019.
- "Approval of Incivek (telaprevir), a direct acting antiviral drug (DAA) to treat hepatitis C (HCV)" (Press release). Food and Drug Administration. 24 May 2011. Retrieved 25 June 2012.
- "FDA approves Kalydeco to treat rare form of cystic fibrosis" (Press release). Food and Drug Administration. 31 January 2011. Retrieved 24 June 2012.
- "KALYDECO™ (ivacaftor) tablets". Vertex Pharmaceuticals. Retrieved 24 June 2012.
- Walker, Joseph; Rockoff, Jonathan D. (19 November 2014). "Cystic Fibrosis Foundation Sells Drug's Rights for $3.3 Billion". Wall Street Journal. ISSN 0099-9660. Retrieved 10 June 2019.
- Herper, Matthew. "For Vertex Pharmaceuticals, Can One Billion-Dollar Breakthrough Beget Another?". Forbes. Retrieved 10 June 2019.
- "VX-809 (Cystic Fibrosis)". Vertex Pharmaceuticals. 7 September 2011. Retrieved 24 June 2012.
- "Vertex Wins Approval for Kalydeco to Treat Cystic Fibrosis". 31 January 2012. Retrieved 10 June 2019.
- "Xconomy: Vertex Seeks FDA Green Light for Cystic Fibrosis Drug, Second Potential Hit of Big Year". Xconomy. 19 October 2011. Retrieved 10 June 2019.
- Deborah Cohen; James Raftery (12 February 2014). "Orphan Drugs: Paying twice: questions over high cost of cystic fibrosis drug developed with charitable funding". BMJ. 348: g1445. doi:10.1136/bmj.g1445.
- Commissioner, Office of the. "Press Announcements - FDA approves new treatment for cystic fibrosis". www.fda.gov. Retrieved 16 January 2017.
- "Is Vertex's New CF Drug Unprecedented? Incremental? Expensive? Maybe All Three". Forbes. 29 March 2017.
- "Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities". Cystic Fibrosis News Today. 17 September 2018.
- "'Just What We Dreamed.' New Vertex Drugs Show Dramatic Benefit Against Cystic Fibrosis". Forbes. 18 July 2017.
- "Vertex, CRISPR Tx Team up on Gene Editing Drugs in $105M Pact". Exome. 26 October 2015.
- "Vertex, Moderna Launch Cystic Fibrosis Collaboration". News: Discovery & Development. Genetic Engineering & Biotechnology News (Paper). 36 (14): 17. August 2016.
- DeAngelis, Allison (3 January 2019). "Vertex teams up with Feng Zhang startup on gene editing tech". www.bizjournals.com. Retrieved 10 June 2019.
- Bloomberg (26 May 2015). "Vertex CEO's $45.8 Million Pay Last Year Excessive, ISS Says". www.bloomberg.com. Retrieved 4 February 2019.
- Reporter, Robert Weisman-. "Vertex CEO's pay falls 38% to $17.4m in second straight decline - The Boston Globe". BostonGlobe.com. Retrieved 3 February 2019.
- Thomas, Katie (24 June 2018). "A Drug Costs $272,000 a Year. Not So Fast, Says New York State". The New York Times. ISSN 0362-4331. Retrieved 3 February 2019.
- Jul 24, Lisa Johnson · CBC News · Posted:; July 25, 2018 7:50 PM PT | Last Updated:; 2018. "Cystic fibrosis patients sue health ministers for coverage of expensive drug for rare disease | CBC News". CBC. Retrieved 3 February 2019.
- editor, Sarah Boseley Health (3 February 2019). "Cystic fibrosis: the patients facing shorter lives due to drug's expense". The Guardian. ISSN 0261-3077. Retrieved 3 February 2019.
- "CF Support Group | Letter to Dr Jeffrey Leiden". CF Support Group. Retrieved 3 February 2019.
- "The problem is the price: 5 reasons the cystic fibrosis community and its allies must band together to speak out about our drug prices – right now". Patients For Affordable Drugs. 17 May 2018. Retrieved 3 February 2019.
- Wnuk, Piotr (18 July 2018). "UK urges Vertex to accept 'generous' Orkambi funding offer". Pharmaphorum. Retrieved 19 June 2019.
- "Cystic fibrosis drug deadlock 'must end'". 4 February 2019. Retrieved 19 June 2019.
- Ramsden, David (16 July 2018). "Just got this and wanted to share now. @NHSEngland have made their "final" offer to @VertexPharma . Headline numbers look big - but deeply concerned that they don't truly reflect the difference the pipeline of drugs will make. Keep talking and get a deal done! #OrkambiNowpic.twitter.com/ECsrTpN7hS". @davidramcft. Retrieved 3 February 2019.
- "Vertex Pharmaceuticals media statement NHS E reimbursement meeting July 4". 5 July 2018.
- "Orkambi: Drugs maker says NHS 'undervalues' patient lives". Sky News. Retrieved 3 February 2019.
- Boseley, Sarah (27 March 2019). "600 years' supply of cystic fibrosis drug destroyed in price row". the Guardian. Retrieved 29 March 2019.
- "Nearly 8,000 Packets of Orkambi Destroyed as the U.K. and Vertex Hash out Pricing Concerns". BioSpace. Retrieved 19 June 2019.
- Deborah, Cohen; Raftery, James (18 February 2014). "Paying twice: the "charitable drug" with a high price tag" (PDF). BMJ. 348: 18–19.
- Andrew Pollack (19 November 2014), Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern, New York Times, retrieved 19 July 2015
- Joseph Walker; Jonathan D. Rockoff (19 November 2014), Cystic Fibrosis Foundation Sells Drug's Rights for $3.3 Billion: The Biggest Royalty Purchase Ever Reflects Group's Share of Kalydeco Sales, Wall Street Journal, retrieved 19 July 2015
- Pollack, Andrew (19 November 2014). "Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern". The New York Times. ISSN 0362-4331. Retrieved 9 July 2019.
- Brady Dennis (2 July 2015), Are risks worth the rewards when nonprofits act like venture capitalists?, Washington Post, retrieved 19 July 2015
- John Fauber (19 May 2013). "Cystic Fibrosis: Charity and Industry Partner for Profit". MedPage Today, Milwaukee Journal Sentinel. Retrieved 19 July 2015.
- David M. Orenstein; Paul M. Quinton; Brian P. O'Sullivan; Carlos E. Milla; Mark Pian; et al. (9 July 2012), Letter to Jeff Leiden, CEO of Vertex Pharmaceuticals (PDF), retrieved 19 July 2015
- "Voices Carry: How History Informs Patient Advocacy in the Biotech Industry", Life Sciences Foundation, Cambridge, Massachusetts, 15 April 2015, archived from the original on 7 March 2016, retrieved 19 July 2015
- "Voices Carry: How History Informs Patient Advocacy in the Biotech Industry". YouTube. 14 May 2014. 1 hour 3 min -- 1 hour 5 min. Retrieved 3 July 2019.
- Annual Report. Vertex Pharmaceuticals. 15 March 2004. Retrieved 24 June 2012.
- "Vertex Completes New Drug Application for Telaprevir for Hepatitis C" (Press release). Vertex Pharmaceuticals. 23 November 2010. Retrieved 24 June 2012.
- Business data for Vertex Pharmaceuticals: