Founded in 1989 in Cambridge, Mass., Vertex Pharmaceuticals today has research and development sites and commercial offices in the United States, Europe, Canada and Australia.[/td_text_with_title]
Orkampi was approved thursday 2nd July 2015 by the FDA. Approval in europe is still pending. The drug costs 259.000 dollars given a sigh of relief to investors, sending Vertex Pharmaceuticals up in thursday and friday session. Well done.
|Industry||Pharmaceuticals & Biotherapeutics)|
|Revenue||US$1.4 billion (2011)|
Vertex Pharmaceuticals, Inc. is an American pharmaceutical company based in Boston, Massachusetts.
Vertex was founded in 1989 by Joshua Boger and Kevin J. Kinsella. Vertex was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry.
By 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer. It maintains headquarters in South Boston, Massachusetts, and two research facilities, in San Diego, California, and Oxford, England. The company's beginnings were profiled by Barry Werth in the 1994 book The Billion-Dollar Molecule and its further development in his 2014 book, The Antidote: Inside the World of New Pharma. In 2009, the company had about 1,800 employees, including 1,200 in the Boston area.
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 will mark the first time in the company's history that all of the roughly 1,200 Vertex employees in the Greater Boston area will be working together.
Since late 2011, when Jeffrey M. Leiden joined Vertex as CEO, 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. Vertex posted only one annual profit since it was founded in 1989.
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 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. About 4% of those, or 1,200, have the G551D gene mutation. Vertex is marketing the drug at $307,000 a year per patient. Vertex also is studying ivacaftor in combination with another drug (lumacaftor) for the most common mutation in CF, known as F508del, and expects the first set of results in 2012. Vertex worked for 13 years with the Cystic Fibrosis Foundation to develop the drug.
In the UK, the company provided the drug free for a limited time for certain patients, then left the hospitals to decide whether to continue to pay for it 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.
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 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.
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."
"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 25% of Cystic Fibrosis patients are not getting the medical care they need because of the prohibitive cost of the drug.
- "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.
- 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
- "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.
- "Kalydeco: A Price Too High to Pay?".
- "VX-809 (Cystic Fibrosis)". Vertex Pharmaceuticals. 7 September 2011. Retrieved 24 June 2012.
- Edney, Anna (2 February 2012). "Vertex Wins Approval for Kalydeco to Treat Cystic Fibrosis". Bloomberg Businessweek. Retrieved 24 June 2012.
- 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 2017-01-16.
- "Vertex, Moderna Launch Cystic Fibrosis Collaboration". News: Discovery & Development. Genetic Engineering & Biotechnology News (Paper). 36 (14): 17. August 2016.
- Andrew Pollack (November 19, 2014), Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern, New York Times, retrieved July 19, 2015
- Joseph Walker; Jonathan D. Rockoff (November 19, 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 July 19, 2015
- Brady Dennis (2 July 2015), Are risks worth the rewards when nonprofits act like venture capitalists?, Washington Post, retrieved 19 July 2015
- John Fauber (May 19, 2013). "Cystic Fibrosis: Charity and Industry Partner for Profit". MedPage Today, Milwaukee Journal Sentinel. Retrieved July 19, 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, April 15, 2015, retrieved July 19, 2015
- 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.