Vertex Pharmaceuticals

Last updated

Vertex Pharmaceuticals Incorporated
Company type Public
Industry
Founded1989;36 years ago (1989)
Headquarters Boston, Massachusetts, U.S.
Key people
Products Pharmaceuticals
RevenueIncrease2.svg US$9.87 billion (2023)
Decrease2.svgUS$3.83 billion (2023)
Increase2.svgUS$3.62 billion (2023)
Total assets Increase2.svgUS$22.7 billion (2023)
Total equity Increase2.svgUS$17.6 billion (2023)
Number of employees
c. 5,400 (2023)
Website vrtx.com
Footnotes /references
[1] [2]

Vertex Pharmaceuticals Incorporated 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 Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.

Contents

History

Vertex was founded in 1989 by Joshua Boger [3] and Kevin J. Kinsella [4] to "transform the way serious diseases are treated." [5]

The company's beginnings were profiled by Barry Werth in the 1994 book The Billion-Dollar Molecule . [4] His 2014 book, The Antidote: Inside the World of New Pharma, chronicled the company's subsequent development over the next two decades. [6]

By 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer. [7]

In 2009, the company had about 1,800 employees, including 1,200 in the Boston area. [3] By 2019 there were about 2,500 employees. [8]

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. [9] 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. [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. [11] 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. [12] [13]

In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash. [14] Semma Therapeutics created a "small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out." [15]

On 1 April 2020, Reshma Kewalramani, then the chief medical officer, became president and chief executive officer of Vertex Pharmaceuticals. [16] Former CEO and president Jeffrey Leiden transitioned to the role of executive chairman of the board of directors, as of 1 April 2020. [16] Leiden was to serve as executive chairman until April 2023, [17] [18] although As of September 2024 he remains in that post. [19]

In July 2022, the business announced it would acquire ViaCyte, Inc. for $320 million. [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. [21]

Medicines

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 [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.

In the US, 30,000 people have cystic fibrosis. About 4% of those, or 1,200, have the G551D gene mutation. In 2017 Vertex marketed the drug for $311,000 per person per year. [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. Vertex produced the drug after 13 years of research and development, with $70 million in support from the Cystic Fibrosis Foundation. [27] [28]

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 [29] 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. [30] 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. [31]

On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis. [32] On 12 February 2018, the FDA approved the combination, marketed as Symdeko. [33]

Triple combination treatments

In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor. [33] In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population. [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. [35] The FDA approval covers approximately 90% of patients with cystic fibrosis. [36]

Vertex has refused to make Trikafta available in developing countries and works to block generic alternatives, making it inaccessible for thousands of patients. [37]

Genetic therapies

Pain management

In January 2025, Vertex became the first drugmaker in 25 years to gain U.S. approval for a new type of pain medicine when the FDA approved Journavx (suzetrigine), a novel non-opioid treatment for moderate to severe acute pain. [44] [45] Journavx was evaluated via two randomized, double-blind, placebo- and active-controlled trials of acute surgical pain. [46]

Historical

Telaprevir

In 2014, Vertex discontinued telaprevir. [47] In May 2011, the Food and Drug Administration (FDA) had approved the drug telaprevir (Incivek) as an oral treatment for hepatitis C. Development and commercialization of telaprevir was shared with Johnson & Johnson for European distribution and Mitsubishi for Asia. Telaprevir is a protease inhibitor. [48]

CEO compensation

In 2015, several media outlets reported CEO Jeffrey Leiden's 2014 compensation to be approximately US$48.5 million. [49] Vertex shareholders opted for a reduction in CEO compensation in 2015 and 2016, resulting in 2016 earnings of US$17.4 million. [50]

Pricing in the UK

The initial pricing of Orkambi at US$ 135,000 to US$ 270,000 per patient per year, depending on the patient's country, led to campaigns by parent groups concerned that it limited access to the drug for children and young adults with cystic fibrosis, and also led to allegations of unfair pricing by the UK's National Health Service. [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. The funding agreement was finalized soon after Vertex signed reimbursement deals elsewhere, including NHS Scotland, Spain and Australia. [58] [36] Vertex agreed to offer equivalent pricing to the NHS in Wales and Northern Ireland . [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. However, in June 2024, NICE and Vertex reached an agreement on acceptable pricing terms, extending the pricing deal originally established in 2019 to include these drugs. The extended agreement also ensures that patients will have access to future license extensions for these CF medications. [61] [62] [63]

Global pricing

Pricing and public funding varies by country beyond the USA and the UK.

By October 2019, Vertex's CF medicines were publicly funded in 17 countries. [64]

By 2023, Vertex had reimbursement agreements in 36 countries for their CF medicines, of which 35 are considered high-income countries. 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. [65]

Trikafta has become increasingly available worldwide since its initial approval in 2019. As of 2023, the medication is reimbursed in over 30 countries, primarily in high-income nations across North America, Europe, and Oceania. [66] By 2024, the company's group of CF medications were "broadly available" in over 60 countries. [67] Although access to Cystic Fibrosis medications is still limited in many countries, as of 2024 about two-thirds of people diagnosed are receiving the treatment around the world. [61] The high cost of Trikafta presents a significant barrier to access. [68] In countries where it is reimbursed, patients typically pay a fraction of this cost, but the financial burden on healthcare systems remains substantial. 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]

As of 2024, a large proportion of diagnosed patients in South America, Asia, and Africa had no subsidies for Trikafta, and until 2024, the therapy was inaccessible in Mexico and Cuba. [65] As of April 2024, Trikafta was made available in South Africa via agreements between Vertex and several commercial insurers to make the medication available to individual patients. [70] As of September 2024, Mexico's Cofepris authorized Trikafta as the first orphan drug for the care of cystic fibrosis patients. [71] [72]

Venture philanthropy

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. [73] 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. [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. [73]

By 2015 the annual price of Kalydeco had been increased to more than $300,000 per patient. [75]

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." [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. 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." [76]

On 15 April 2015 in Cambridge, Massachusetts, 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. [78] While Brooks, who has cystic fibrosis, expressed gratitude for Vertex's development of Kalydeco, she observed that "More than 25% of people are saying (in 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." [79]

See also

Related Research Articles

<span class="mw-page-title-main">Cystic fibrosis</span> Genetic disorder affecting mostly the lungs

Cystic fibrosis (CF) is a genetic disorder inherited in an autosomal recessive manner that impairs the normal clearance of mucus from the lungs, which facilitates the colonization and infection of the lungs by bacteria, notably Staphylococcus aureus. CF is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. The hallmark feature of CF is the accumulation of thick mucus in different organs. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms may include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in most males. Different people may have different degrees of symptoms.

In genetics, a nonsense mutation is a point mutation in a sequence of DNA that results in a nonsense codon, or a premature stop codon in the transcribed mRNA, and leads to a truncated, incomplete, and possibly nonfunctional protein product. Nonsense mutations are not always harmful; the functional effect of a nonsense mutation depends on many aspects, such as the location of the stop codon within the coding DNA. For example, the effect of a nonsense mutation depends on the proximity of the nonsense mutation to the original stop codon, and the degree to which functional subdomains of the protein are affected. As nonsense mutations leads to premature termination of polypeptide chains; they are also called chain termination mutations.

<span class="mw-page-title-main">Gilead Sciences</span> American pharmaceutical company

Gilead Sciences, Inc. is an American biopharmaceutical company headquartered in Foster City, California, that focuses on researching and developing antiviral drugs used in the treatment of HIV/AIDS, hepatitis B, hepatitis C, influenza, and COVID-19, including ledipasvir/sofosbuvir and sofosbuvir. Gilead is a member of the Nasdaq-100 and the S&P 100.

<span class="mw-page-title-main">Cystic fibrosis transmembrane conductance regulator</span> Mammalian protein found in humans

Cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel in vertebrates that is encoded by the CFTR gene.

<span class="mw-page-title-main">Cystic Fibrosis Foundation</span> American non-profit organisation

The Cystic Fibrosis Foundation (CFF) is a 501(c)(3) non-profit organization in the United States established to provide the means to cure cystic fibrosis (CF) and ensure that those living with CF live long and productive lives. The Foundation provides information about cystic fibrosis and finances CF research that aims to improve the quality of life for people with the disease. The Foundation also engages in legislative lobbying for cystic fibrosis.

Venture philanthropy is a type of impact investment that takes concepts and techniques from venture capital finance and business management and applies them to achieving philanthropic goals. The term was first used in 1969 by John D. Rockefeller III to describe an imaginative and risk-taking approach to philanthropy that may be undertaken by charitable organizations.

<span class="mw-page-title-main">Ataluren</span> Duchenne muscular dystrophy medication

Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics.

<span class="mw-page-title-main">Telaprevir</span> Chemical compound

Telaprevir (VX-950), marketed under the brand names Incivek and Incivo, is a pharmaceutical drug for the treatment of hepatitis C co-developed by Vertex Pharmaceuticals and Johnson & Johnson. It is a member of a class of antiviral drugs known as protease inhibitors. Specifically, telaprevir inhibits the hepatitis C viral enzyme NS3/4A serine protease. Telaprevir is only indicated for use against hepatitis C genotype 1 viral infections and has not been proven to be safe or effective when used for other genotypes of the virus. The standard therapy of pegylated interferon and ribavirin is less effective than telaprevir in those with genotype 1.

<span class="mw-page-title-main">Ivacaftor</span> Cystic fibrosis treatment drug

Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, who account for 4–5% cases of cystic fibrosis. It is also included in combination medications, lumacaftor/ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor which are used to treat people with cystic fibrosis.

Lumacaftor (VX-809) is a pharmaceutical drug that acts as a chaperone during protein folding and increases the number of CFTR proteins that are trafficked to the cell surface. It is available in a single pill with ivacaftor; the combination, lumacaftor/ivacaftor, is used to treat people with cystic fibrosis who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the defective protein that causes the disease. It was developed by Vertex Pharmaceuticals and the combination was approved by the FDA in 2015. As of 2015, lumacaftor had no medical use on its own.

<span class="mw-page-title-main">AbbVie</span> American pharmaceutical company

AbbVie Inc. is an American pharmaceutical company headquartered in North Chicago, Illinois. It is ranked sixth on the list of largest biomedical companies by revenue. In 2023, the company's seat in Forbes Global 2000 was 74, and rank 89 on the 2024 list. The company's primary product is Humira (adalimumab), administered via injection. It is approved to treat autoimmune diseases including rheumatoid arthritis, Crohn's disease, plaque psoriasis, and ulcerative colitis.

<span class="mw-page-title-main">Lumacaftor/ivacaftor</span> Cystic fibrosis drug

Lumacaftor/ivacaftor, sold under the brand name Orkambi among others, is a combination of lumacaftor and ivacaftor used to treat people with cystic fibrosis who have two copies of the F508del mutation. It is unclear if it is useful in cystic fibrosis due to other causes. It is taken by mouth.

<span class="mw-page-title-main">Joshua Boger</span> Organic chemist and the founder of Vertex Pharmaceuticals

Joshua S. Boger is an organic chemist and the founder of Vertex Pharmaceuticals Incorporated. He is considered a pioneer in the field of structure-based rational drug design. Drugs developed include amprenavir, an HIV protease inhibitor; telaprevir, a protease inhibitor for treatment of hepatitis C; and Kalydeco, for the treatment of cystic fibrosis. In 2003, Vertex was listed as one of forty worldwide Technology Pioneers by the World Economic Forum. As of 2012, Boger became executive chairman of Alkeus Pharmaceuticals.

<span class="mw-page-title-main">Tezacaftor</span> Chemical compound

Tezacaftor is a medication used for the treatment of cystic fibrosis. It is available in fixed-dose combination medications.

<span class="mw-page-title-main">Jeffrey Leiden</span> CEO of biotechnology company

Jeffrey Leiden is an American physician, scientist and businessman who is the executive chairman of Vertex Pharmaceuticals, a biotechnology company based in Boston, Massachusetts. He was initially appointed to the board of directors of the company in 2009 and was CEO and president from February 2012 to March 2020.

Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta and Kaftrio, is a fixed-dose combination medication used to treat cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor is composed of a combination of ivacaftor, a chloride channel opener, and elexacaftor and tezacaftor, CFTR modulators.

<span class="mw-page-title-main">Elexacaftor</span> Cystic fibrosis medication

Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector.

Peter Grootenhuis was a Dutch-American Medicinal Chemist. Grootenhuis was the Project Leader and Co-Inventor of Ivacaftor (VX-770), the first CFTR potentiator FDA approved drug to treat the underlying cause of Cystic Fibrosis (CF) in patients with certain mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, who account for 4-5% of CF cases. Grootenhuis also led the Vertex team to subsequent discovery of Orkambi, the combination of Ivacaftor and Lumacaftor(VX-809), approved to treat CF in people with two copies of the F508del mutation. Most recently, Grootenhuis's team discovered Tezacaftor (VX-661) and Elexacaftor (VX-445), which in combination with Ivacaftor are the components of Trikafta, a drug approved by the FDA in 2019 to treat CF in more than 90% of CF patients. For Grootenhuis’ contributions to the discovery of these compounds, he was awarded the 2018 IUPAC Richter Prize, the American Chemical Society’s 2013 Heroes of Chemistry Award, and inducted into the American Chemical Society Division of Medicinal Chemistry Hall of Fame. Grootenhuis has contributed to the discovery of over 11 clinical candidates, co-authored more than 100 peer reviewed papers and is inventor of 65 + U.S Patents, and more than 50 EU Patents.

Paul Adrian Negulescu is an American–Romanian cell biologist. He is a Senior Vice President at American pharmaceutical company Vertex Pharmaceuticals. He received the 2022 Shaw Prize in Life science and medicine, together with Michael J. Welsh, for their work that uncovered the etiology of cystic fibrosis and developed effective medications.

<span class="mw-page-title-main">Vanzacaftor/tezacaftor/deutivacaftor</span> Combination medication

Vanzacaftor/tezacaftor/deutivacaftor, sold under the brand name Alyftrek, is a fixed-dose combination medication used for the treatment of cystic fibrosis. It is a combination of deutivacaftor, a CFTR potentiator; tezacaftor; and vanzacaftor, as the calcium salt, vanzacaftor calcium dihydrate. It is taken by mouth.

References

  1. ↑ "Vertex Pharmaceuticals Inc. 2023 Annual Report (Form 10-K)". U.S. Securities and Exchange Commission. 15 February 2024.
  2. ↑ "Vertex Pharmaceuticals Incorporated". Hoover's . Retrieved 24 June 2012.
  3. 1 2 Weisman, Robert (24 April 2011). "Rearranging the Corporate DNA". Boston Sunday Globe . p. G1. Retrieved 25 June 2012.
  4. 1 2 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.
  5. ↑ Roth, Michael S. (21 May 2011). "Why liberal arts matter". CNN . Retrieved 7 January 2022.
  6. ↑ Werth, Barry (2014). The Antidote: Inside the World of New Pharma. New York: Simon & Schuster. ISBN   9781451655667. OCLC   859375019.
  7. ↑ "Vertex Pharmaceuticals Reports Fourth Quarter and Full Year 2004 Financial Results and Full Year 2005 Guidance". Vertex Pharmaceuticals. Retrieved 30 May 2019.
  8. ↑ "Vertex Pharmaceuticals on the Forbes Global 2000 List". Forbes. Retrieved 30 May 2019.
  9. ↑ 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
  10. ↑ Weisman, Robert (4 February 2014). "New complex unites Vertex employees". The Boston Globe. Retrieved 30 May 2019.
  11. ↑ Owens, Jeremy C. (23 January 2019). "Vertex terminates COO for 'personal behavior'". MarketWatch . Retrieved 25 January 2019.
  12. ↑ "Vertex Expands into New Disease Areas and Enhances Gene Editing Capabilities Through Expanded Collaboration with CRISPR Therapeutics and Acquisition of Exonics Therapeutics". BioSpace.
  13. ↑ "Vertex Dives into DMD with Exonics Acquisition and CRISPR Therapeutics Partnership Expansion". BioSpace. 7 June 2019.
  14. ↑ "Vertex to buy cell therapy developer Semma for $950 million". 3 September 2019. Archived from the original on 9 July 2020 – via uk.reuters.com.
  15. ↑ Time Staff. "12 Innovations That Will Change Health Care and Medicine in the 2020s". Time. Retrieved 29 October 2019.
  16. 1 2 DeAngelis, Allison (2 April 2020). "VERTEX 3.0: One of the area's largest biotechs enters new era of leadership, drug development". www.bizjournals.com. Retrieved 6 April 2020.
  17. ↑ "Vertex's Reshma Kewalramani takes CEO post as longtime vet Jeff Leiden moves to new role". Endpoints News. Retrieved 31 July 2019.
  18. ↑ "Vertex Pharmaceuticals' Jeffrey Leiden to step aside as CEO". STAT. 25 July 2019. Retrieved 31 July 2019.
  19. ↑ "Jeffrey Leiden, M.D., Ph.D." Vertex.
  20. ↑ "Vertex to Acquire ViaCyte, with the Goal of Accelerating its Potentially Curative VX-880 Programs in Type 1 Diabetes". 11 July 2022.
  21. ↑ "Vertex to acquire Alpine Immune Sciences for $4.9bn - Pharmafile". pharmafile.com. 11 April 2024. Retrieved 11 April 2024.
  22. ↑ "FDA approves Kalydeco to treat rare form of cystic fibrosis" (Press release). Food and Drug Administration. 31 January 2011. Retrieved 24 June 2012.
  23. ↑ "KALYDECO™ (ivacaftor) tablets". Vertex Pharmaceuticals. Retrieved 24 June 2012.
  24. ↑ Walker, Joseph; Rockoff, Jonathan D. (19 November 2014). "Cystic Fibrosis Foundation Sells Drug's Rights for $3.3 Billion". The Wall Street Journal. ISSN   0099-9660 . Retrieved 10 June 2019.
  25. ↑ Herper, Matthew. "For Vertex Pharmaceuticals, Can One Billion-Dollar Breakthrough Beget Another?". Forbes. Retrieved 10 June 2019.
  26. ↑ "VX-809 (Cystic Fibrosis)". Vertex Pharmaceuticals. 7 September 2011. Retrieved 24 June 2012.
  27. ↑ "Vertex Wins Approval for Kalydeco to Treat Cystic Fibrosis". 31 January 2012. Retrieved 10 June 2019.
  28. ↑ "Xconomy: Vertex Seeks FDA Green Light for Cystic Fibrosis Drug, Second Potential Hit of Big Year". Xconomy. 19 October 2011. Retrieved 10 June 2019.
  29. ↑ 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 (feb12 1): g1445. doi:10.1136/bmj.g1445. PMID   24523379. S2CID   8702814.
  30. ↑ "Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation | Vertex Pharmaceuticals". investors.vrtx.com.
  31. ↑ "Press Announcements – FDA approves new treatment for cystic fibrosis". www.fda.gov (Press release). Office of the Commissioner. Retrieved 16 January 2017.
  32. ↑ "Is Vertex's New CF Drug Unprecedented? Incremental? Expensive? Maybe All Three". Forbes. 29 March 2017.
  33. 1 2 "Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities". Cystic Fibrosis News Today. 17 September 2018.
  34. ↑ "'Just What We Dreamed.' New Vertex Drugs Show Dramatic Benefit Against Cystic Fibrosis". Forbes . 18 July 2017.
  35. ↑ "FDA approves new breakthrough therapy for cystic fibrosis". FDA. 21 October 2019.
  36. 1 2 Parsons, Lucy (24 October 2019). "Vertex, NHS England and NICE finally reach agreement for Orkambi". PMLive. Retrieved 29 October 2019. 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.
  37. ↑ Nolen, Stephanie; Robbins, Rebecca (7 February 2023). "The Drug Is a 'Miracle' but These Families Can't Get It - Vertex Pharmaceuticals is not making its drug, Trikafta, available in poorer countries, where thousands of diagnosed patients stand to benefit". The New York Times . ISSN   0362-4331 . Retrieved 29 March 2023.
  38. ↑ "Vertex, CRISPR Tx Team up on Gene Editing Drugs in $105M Pact". Exome. 26 October 2015.
  39. ↑ CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001™ for the Treatment of Severe Hemoglobinopathies, PM Vertex May 11, 2020; retrieved May 11, 2020
  40. ↑ "Vertex, Moderna Launch Cystic Fibrosis Collaboration". News: Discovery & Development. Genetic Engineering & Biotechnology News (Paper). 36 (14): 17. August 2016.
  41. 1 2 Speights, Keith; Orelli, Brian (4 September 2021). "Why Vertex Is Expanding a Gene-Editing Partnership -- and It's not With CRISPR Therapeutics". nasdaq.com. Retrieved 12 September 2021.
  42. ↑ DeAngelis, Allison (3 January 2019). "Vertex teams up with Feng Zhang startup on gene editing tech". www.bizjournals.com. Retrieved 10 June 2019.
  43. ↑ "Pipeline | Vertex Pharmaceuticals". www.vrtx.com.
  44. ↑ Constantino, Annika Kim (30 January 2025). "FDA approves Vertex's non-opioid painkiller, first new kind of pain medicine in decades". CNBC. Retrieved 2 February 2025.
  45. ↑ Goodman, Brenda (30 January 2025). "FDA approves first new type of pain medication in 25 years". CNN. Retrieved 2 February 2025.
  46. ↑ Commissioner, Office of the (30 January 2025). "FDA Approves Novel Non-Opioid Treatment for Moderate to Severe Acute Pain". FDA. Retrieved 2 February 2025.
  47. ↑ Silverman, Ed (12 August 2014). "From Riches to Rags: Vertex Discontinues Incivek as Sales Evaporate". Pharmalot (Blog). Dow Jones & Company. Retrieved 13 August 2014.
  48. ↑ "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.
  49. ↑ Bloomberg (26 May 2015). "Vertex CEO's $45.8 Million Pay Last Year Excessive, ISS Says". www.bloomberg.com. Retrieved 4 February 2019.
  50. ↑ Weisman, Robert (17 April 2017). "Vertex CEO's pay falls 38% to $17.4m in second straight decline". The Boston Globe. Retrieved 3 February 2019.
  51. ↑ 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.
  52. ↑ Johnson, Lisa (25 July 2018). "Cystic fibrosis patients sue health ministers for coverage of expensive drug for rare disease". CBC News. Retrieved 3 February 2019.
  53. ↑ Boseley, Sarah (3 February 2019). "Cystic fibrosis: the patients facing shorter lives due to drug's expense". The Guardian. ISSN   0261-3077 . Retrieved 3 February 2019.
  54. ↑ "CF Support Group | Letter to Dr Jeffrey Leiden". CF Support Group. Retrieved 3 February 2019.
  55. ↑ "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.
  56. ↑ Boseley, Sarah (27 March 2019). "600 years' supply of cystic fibrosis drug destroyed in price row". the Guardian. Retrieved 29 March 2019.
  57. ↑ "Nearly 8,000 Packets of Orkambi Destroyed as the U.K. and Vertex Hash out Pricing Concerns". BioSpace. 28 March 2019. Retrieved 19 June 2019.
  58. ↑ Silverman, Ed (24 October 2019). "Vertex and the U.K. reach a deal for cystic fibrosis medicines". STAT. Retrieved 29 October 2019.
  59. ↑ "NHS England finally strikes deal for high value drug". Health Service Journal. 24 October 2019. Retrieved 3 December 2019.
  60. ↑ MS, Marisa Wexler (1 July 2020). "NHS, Vertex Strike Deal to Make Kaftrio Available in England". Cystic Fibrosis News Today. Retrieved 9 July 2020.
  61. 1 2 Manalac, Tristan (21 June 2024). "Vertex Finally Reaches Pricing Deal With England's NHS for Cystic Fibrosis Drugs". BioSpace.
  62. ↑ Furlong, Ashleigh (20 June 2024). "Vertex Reaches Pricing Deal With NHS for Cystic Fibrosis Drugs". Bloomberg.
  63. ↑ Slawther, Eliza (20 June 2024). "England's NICE Backs Vertex's CF Drugs Kaftrio, Symkevi & Orkambi After Initial Rejection". Pink Sheet.
  64. ↑ "Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor)". www.businesswire.com. 21 October 2019.
  65. 1 2 Lobo, Andrea (26 April 2024). "Access to Trikafta almost exclusive to high-income countries: Study". Cystic Firbrosis News Today.
  66. ↑ Inácio P (22 February 2022). "CF More Prevalent Worldwide Than Thought, Access to Trikafta Limited". Cystic Firbrosis News Today.
  67. ↑ Kansteiner, Fraiser (21 June 2024). "Vertex clinches Trikafta and other cystic fibrosis drug reimbursement deal in England". FiercePharma.
  68. ↑ Kansteiner F (14 February 2022). "Vertex's Trikafta pricing central to cystic fibrosis treatment disparities, study says".
  69. ↑ Abdallah K, De Boeck K, Dooms M, Simoens S (2021). "A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe". Frontiers in Pharmacology. 12: 746710. doi: 10.3389/fphar.2021.746710 . PMC   8630624 . PMID   34858177.
  70. ↑ Silverman, Ed (20 April 2024). "Vertex offers limited access to a cystic fibrosis drug in South Africa — and may undercut a court case". STAT.
  71. ↑ "México, uno de los primeros países en autorizar tratamiento para fibrosis quística". Gobierno de Mexico. 26 September 2024.
  72. ↑ Montesinos, Patricia (3 October 2024). "Cofepris authorizes medical registration of drug for cystic fibrosis". Canal 13 Mexico.
  73. 1 2 3 Andrew Pollack (19 November 2014), "Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern", The New York Times, retrieved 19 July 2015
  74. ↑ 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, The Wall Street Journal, retrieved 19 July 2015
  75. ↑ Brady Dennis (2 July 2015), "Are risks worth the rewards when nonprofits act like venture capitalists?", The Washington Post, retrieved 19 July 2015
  76. 1 2 John Fauber (19 May 2013). "Cystic Fibrosis: Charity and Industry Partner for Profit". MedPage Today, Milwaukee Journal Sentinel. Retrieved 19 July 2015.
  77. ↑ 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
  78. ↑ "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
  79. ↑ "Voices Carry: How History Informs Patient Advocacy in the Biotech Industry". YouTube. 14 May 2014. 1 hour 3 min – 1 hour 5 min. Archived from the original on 13 December 2021. Retrieved 3 July 2019.
This page is based on this Wikipedia article
Text is available under the CC BY-SA 4.0 license; additional terms may apply.
Images, videos and audio are available under their respective licenses.