Вертекс википедия компания фармацевтическая

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Vertex Pharmaceuticals Incorporated

Type	Public company
Traded as	Nasdaq: VRTX Nasdaq-100 component S&P 500 component
Industry	Pharmaceuticals & Biotherapeutics[1]
Founded	1989; 34 years ago
Headquarters	Boston, Massachusetts, U.S.
Key people	Reshma Kewalramani, M.D., president and CEO Stuart A. Arbuckle, executive vice president and chief commercial officer Michael J. Parini, executive vice president and chief legal, administrative and business development officer Jeffrey Leiden, M.D., PhD, executive chairman David Altshuler, M.D., PhD, executive vice president, global research and chief scientific officer Amit K. Sachdev, executive vice president and chief patient officer
Products	Pharmaceuticals
Revenue	US$8.93 billion (2022)
Number of employees	4,500
Website	www.vrtx.com

Vertex Pharmaceuticals 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, Oxfordshire, England.

History[edit]

Vertex was founded in 1989 by Joshua Boger^[2] and Kevin J. Kinsella^[3] to «transform the way serious diseases are treated.»^[4]

The company’s beginnings were profiled by Barry Werth in the 1994 book, The Billion-Dollar Molecule.^[3] His 2014 book, The Antidote: Inside the World of New Pharma, chronicled the company’s subsequent development over the next two decades.^[5]

By 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer.^[6]

In 2009, the company had about 1,800 employees, including 1,200 in the Boston area.^[2] By 2019 there were about 2,500 employees.^[7]

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.^[8] 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.^[9]

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.^[10] 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.^[11][12]

In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash.^[13] Semma Therapeutics created a «small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out.»^[14]

On 1 April 2020, former chief medical officer, Reshma Kewalramani, became president and chief executive officer of Vertex Pharmaceuticals.^[15] Former CEO and president Jeffrey Leiden transitioned to the role of executive chairman of the board of directors, as of 1 April 2020.^[15] Dr. Leiden will serve as executive chairman until April 2023.^[16][17]

In July 2022, the business announced it would acquire ViaCyte, Inc. for $320 million.^[18]

Medicines[edit]

Ivacaftor, lumacaftor and tezacaftor[edit]

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^[19] of the first drug, Kalydeco,^[20] 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 year.^[21][22]

Vertex also studied ivacaftor in combination with another drug (lumacaftor^[23]) 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.^[24][25]

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^[26] 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.^[27] 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.^[28]

On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis.^[29] On 12 February 2018, the FDA approved the combination, marketed as Symdeko.^[30]

Triple combination treatments[edit]

In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor.^[30] In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population.^[31]
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 new drug application was filed.^[32] Trikafta is approved for approximately 90% of patients with cystic fibrosis.^[33]

Vertex has refused to make Trikafta available in developing countries and works to block generic alternatives, making it inaccessible for thousands of patients.^[34]

Genetic therapies[edit]

• In 2015, Vertex entered into a research collaboration with CRISPR Therapeutics to develop gene-editing therapies for genetic diseases.^[35] 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.^[36]
• 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.^[37]
• In 2019, Vertex established a research collaboration with a privately held company Arbor Biotechnologies^[38] to discover novel proteins to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases.^[39] With seven different areas of focus in the deal, Vertex is paying up to $1.2 billion in milestones along with undisclosed up-front payments.^[38]
• Vertex’s product pipeline includes drug regimens targeting specialty diseases like sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, alpha-1 antitrypsin deficiency, pain, and APOL1-mediated kidney diseases.^[40]

Historical[edit]

Telaprevir[edit]

In 2014, Vertex discontinued telaprevir.^[41] 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.^[42]

CEO compensation[edit]

In 2015, several media outlets reported CEO Jeffrey Leiden’s 2014 compensation to be approximately US$48.5 million.^[43] Vertex shareholders opted for a reduction in CEO compensation in 2015 and 2016, resulting in 2016 earnings of US$17.4 million.^[44]

Pricing in the UK[edit]

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.^{[45][46][47][48][49]}

In March 2019, Vertex was legally required to destroy 7,880 packs of Orkambi that reached their expiry dates during negotiations with the NHS concerning the drug’s pricing.^[50][51]

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.^[52][33] The NHS in Wales and Northern Ireland will be offered equivalent pricing terms.^[53] On June 30, 2020, Vertex and NHS expanded the agreement to include reimbursement of Kaftrio, making CF patients in England to be among the first in Europe to access public funding for this medicine upon its approval by the European Commission.^[54]

By October 2019, Vertex’s CF medicines were publicly funded in 17 countries.^[55]

Venture philanthropy[edit]

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.^[56] 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.^[56][57] 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.^[56]

By 2015 the annual price of Kalydeco had been increased to more than $300,000 per patient.^[58]

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.»^[59]

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.^[60]

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.»^[59]

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.^[61] 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.»^[62]

See also[edit]

• Cystic Fibrosis Foundation
• Telaprevir

References[edit]

• Annual Report. Vertex Pharmaceuticals. 15 March 2004. Archived from the original on 3 March 2016. 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.

External links[edit]

• Official website
• Business data for Vertex Pharmaceuticals:
  • Google
  • SEC filings
  • Yahoo!

Генеральный директор ЗАО «ВЕРТЕКС» Георгий Побелянский рассказал корреспонденту «МА» о новых проектах, отношении к санкциям и надежде на развенчание одного устойчивого мифа.

Георгий Эдуардович, «ВЕРТЕКС» существует более 10 лет. Помимо увеличения количества выпускаемых препаратов, что означает для компании этот срок? В чем принципиальное отличие в работе сегодня и тогда, в начале деятельности?
— В июле 2014 г. компания отметила 11-летие с момента получения первой лицензии на производство лекарств. В декабре мы отпразднуем 15 лет со дня ее основания. По меркам развития новейшей истории фармрынка — это действительно солидный срок. За этот период «ВЕРТЕКС» прошел путь от небольшого предприятия до производителя лекарств, косметики и БАД с собственной научно-исследовательской базой. Чтобы увеличить ассортимент, надо было обеспечить возможности для этого процесса. Развитие портфеля невозможно без наращивания мощностей и парка современного оборудования, работы высококвалифицированных специалистов и расширения их штата, грамотной маркетинговой стратегии компании и организации сбыта продуктов.

Сегодня в компании «ВЕРТЕКС» работают 13 кандидатов химических, технических, медицинских, биологических, фармацевтических наук. Дистрибуция нашей продукции охватывает всю территорию России, часть косметического ассортимента представлена также в Беларуси и Казахстане. Мы реализуем проект по строительству крупного инновационно-производственного комплекса в особой экономической зоне в Санкт-Петербурге. На данный момент завершено строительство здания первой очереди завода, ведется установка оборудования, оснащаются «чистые» и производственные помещения. То есть развитие компании не прекращается. Мы растем вместе с рынком и реагируем на его условия и изменения, предлагая самый актуальный ассортимент. Когда «ВЕРТЕКС» создавался, была другая законодательная база, другие требования к производству и обеспечению качества продукции. Они совершенствуются, и мы, в свою очередь, обязаны их соблюдать.

Отмечу, что выпуск средств под собственными брендами, создание торговых марок косметических линеек планировались сразу. Поэтому постепенно в компании создавались служба науки и служба разработки. Не стоит забывать о зависимости между производством дженериков и инвестированием прибыли от их продаж в разработку инновационных оригинальных препаратов. Экономически целесообразно делать это постепенно, накопив интеллектуальный и финансовый капитал, то есть создать базу для собственных разработок. Что мы и сделали. Развитие ассортимента — постоянный процесс. Сейчас в портфеле компании более 160 позиций лекарственных, косметических средств и БАД. Столько же находится в разработке. Удвоить количество наименований в ассортименте мы планируем к 2019 г.

Расскажите, пожалуйста, о трех оригинальных препаратах, исследования над которыми ведутся сейчас. Из какой они терапевтической области?
— Наша служба науки проводит разработку трех новых оригинальных препаратов, перспективных для лечения широко распространенных социально значимых заболеваний в кардиологии и неврологии. Над этими проектами мы трудимся с 2007 г., разработки уже защищены рядом патентов. Один из проектов надеемся завершить в 2018 г. Период проведения доклинических исследований давно окончен, по их итогам препараты доказали свою эффективность и безопасность для продолжения исследований.

Компания строит завод в ОЭЗ «Санкт-Петербург». В чем инновационность строящегося производства?
— Само участие в проекте особых экономических зон предполагает инновационный подход к производству и формированию продуктового портфеля компании, обеспечение современных стандартов качества и безопасности процесса выпуска продукции. Комплекс спроектирован в соответствии с требованиями стандарта надлежащей производственной практики GMP. Для функционирования помещений и мощностей устанавливается самое современное автоматизированное оборудование.

Реализация масштабного проекта по строительству комплекса связана с планами по развитию продуктового портфеля компании. Инновационность ассортимента, прежде всего, заключается в разработке, внедрении в производство и в дальнейшем выводе на рынок оригинальных лекарственных препаратов. Помимо производственного на заводе будет располагаться лабораторный корпус, где мы продолжим разработку и научные исследования своих продуктов, создав для этого самые благоприятные условия.

Вы ориентируетесь на стандарты GMP. Хватает ли компании кадров, ориентированных для работы по стандартам? Есть ли программы подготовки специалистов?
— В функционировании системы управления качеством задействовано более 100 сотрудников компании. Расширение номенклатуры и строительство завода требуют новых кадров. Активно растет штат отдела валидации в связи с большим объемом соответствующих работ.

На регулярной основе организовано обучение специалистов, чья деятельность связана с качеством, включая требования GMP. Они повышают свою квалификацию в лицензированных внешних организациях, в частности в СПХФА, Первом МГМУ им. И.М. Сеченова, в биофармацевтическом кластере «Северный» на базе МФТИ и других. Подготовка руководителей, специалистов и производственных сотрудников проводится также по собственным программам и методическим материалам. Молодые кадры со знанием стандарта приходят в компанию после окончания перечисленных выше вузов, а также РНИМУ им. Н.И. Пирогова и МГОГИ.

В компании идет разработка собственных квазиоригинальных препаратов. Что имеется в виду?
— Мы проводим исследования нескольких собственных квазиоригинальных препаратов, то есть имеющих усовершенствованный состав: в основе каждого – известная молекула, доказавшая свою эффективность, но компоненты лекарства применяются в новой комбинации, имеют не использовавшуюся ранее форму выпуска или предполагают новый способ приема препарата, например, обеспечивая его пролонгированное действие. Лекарства будут применяться для лечения заболеваний в кардиологии, ЛОР, гинекологии и дерматологии. Наши научные разработки в этой области также защищены рядом патентов. Выпуск первого из таких препаратов запланирован на 2015 г.

Стратегия развития фармпромышленности в РФ «Фарма-2020» нацелена в том числе на импортозамещение эффективными отечественными средствами. Какую продукцию в этом плане готова предложить компания «ВЕРТЕКС»? Особенно сегодня, когда страна живет в режиме санкций со стороны Запада и США.
— «ВЕРТЕКС» — компания со стопроцентным российским капиталом, поэтому весь наш лекарственный ассортимент — более 100 позиций — изначально отвечает требованиям Стратегии. Мы совершенствуем, обновляем, расширяем портфель, отслеживаем сроки действия патентов оригинальных препаратов, охватываем новые для себя направления выпуска продукции с точки зрения ее эффективности и востребованности. При выборе потенциальных для ассортимента позиций рассматриваем самые современные. Помимо этого мы делаем упор на доступность продуктов и результат применения, не уступающий действию дорогого оригинального препарата. Таким образом, мы выполняем социальную функцию и несем ответственность за поддержание здоровья граждан нашей страны. При этом как отечественный производитель и от государства мы хотели бы ответного шага. Например, облегченную регистрацию лекарств, более низкие пошлины и пр.

Не стоит забывать, что «Фарма-2020» направлена также на стимуляцию разработки и производства инновационных препаратов, а также гармонизацию российских стандартов разработки и производства лекарств с международными требованиями. Этим пунктам документа деятельность компании «ВЕРТЕКС» также соответствует.

Ответные санкции Правительства РФ на импорт определенных видов продукции — вынужденная политическая мера. В целом снижение импортозависимости в фармотрасли оцениваем положительно, и не только во время режима санкций: это одна из целей «Фармы-2020» на ближайшие годы. Мы надеемся, что роль и значимость российского производителя, который уже научился соответствовать всем современным требованиям и делать продукты ничуть не хуже иностранных, усилится. Но здесь главное — не перегнуть палку. Например, некоторые производители могут столкнуться с запретом на поставки определенных видов сырья для своих продуктов. А прежде чем их произвести, надо иметь то, из чего они должны быть сделаны.

Насколько возможно, на Ваш взгляд, ограничение импорта иностранной фармпродукции, о чем недавно высказывались в Совете Федерации?
— По информации СМИ, представитель Совета Федерации Николай Журавлев заявлял о необходимости изучить целесообразность ограничения поставок безрецептурных лекарств, у которых есть российские аналоги. В свою очередь, комментируя предложение сенатора, советник Дмитрия Медведева Геннадий Онищенко заверил, что ответные правительственные санкции не коснутся лекарств иностранного производства. Если эта позиция будет пересмотрена, то ограничение импорта фармпродукции надо вводить очень аккуратно, так как степень импортозависимости в стране очень велика, и далеко не все лекарственные препараты, особенно оригинальные с действующей патентной защитой, производятся в России. Необдуманно принятые меры могут лишить российских пациентов доступа к современным лекарственным средствам. Это касается также и биотехнологических препаратов, производство которых сложное, и отечественные предприятия только начинают его осваивать.

При этом следовало бы давать больше преференций отечественным производителям в тех случаях, когда выпуск отечественного аналога оригинального лекарства уже налажен. В целом, я думаю, вопрос должен решаться не ограничениями на импорт фармпродукции, а созданием приоритетных условий для отечественных производителей, в том числе и в госзакупках лекарств.

Часть новинок возглавляемой Вами компании входит в перечень ЖНВЛП. Сейчас Минздрав России работает над пересмотром и расширением списка. Готова ли компания работать и с новым (возможным) списком ЖНВЛП?
— Речь не только о новинках. Около трети портфеля компании и почти половина лекарственного ассортимента входят в перечень ЖНВЛП. С ростом количества средств, которые мы производим, будет увеличиваться и доля жизненно необходимых препаратов — то есть самых нужных для здоровья людей.

Каковы Ваши прогнозы развития отечественного фармрынка на ближайшие несколько лет? Какие основные тренды Вы выделили бы?
— Прежде всего, это рост доли фармпродукции отечественного производства во всех возможных сегментах. Также будет расти спрос на препараты, которые не входят в перечень ЖНВЛП, то есть лекарственные средства не первого выбора, на фармпродукцию для профилактики заболеваний, улучшения внешнего вида (товары для красоты) и пр.

Индустрия ориентируется на международный опыт, формирует новые стандарты и требования к организации производства, к эффективности и качеству продукции. Соответствие фармпроизводств требованиям GMP будет способствовать росту доверия российских потребителей к качеству и эффективности отечественных ЛС. Будем надеяться на долгожданное развенчание мифа, что купленные за границей препараты лучше отечественных современных дженериков.

Планируется создание единого рынка лекарственных средств и медизделий России, Белоруссии, Казахстана и, по последним данным, возможно, еще и Киргизии с 2016 г. Поэтому «ВЕРТЕКС», как и саму фармотрасль, вероятно, ждет много изменений.

From Wikipedia, the free encyclopedia

Vertex Pharmaceuticals Incorporated

Type	Public company
Traded as	Nasdaq: VRTX Nasdaq-100 component S&P 500 component
Industry	Pharmaceuticals & Biotherapeutics[1]
Founded	1989; 34 years ago
Headquarters	Boston, Massachusetts, U.S.
Key people	Reshma Kewalramani, M.D., president and CEO Stuart A. Arbuckle, executive vice president and chief commercial officer Michael J. Parini, executive vice president and chief legal, administrative and business development officer Jeffrey Leiden, M.D., PhD, executive chairman David Altshuler, M.D., PhD, executive vice president, global research and chief scientific officer Amit K. Sachdev, executive vice president and chief patient officer
Products	Pharmaceuticals
Revenue	US$8.93 billion (2022)
Number of employees	4,500
Website	www.vrtx.com

Vertex Pharmaceuticals 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, Oxfordshire, England.

History[edit]

Vertex was founded in 1989 by Joshua Boger^[2] and Kevin J. Kinsella^[3] to «transform the way serious diseases are treated.»^[4]

The company’s beginnings were profiled by Barry Werth in the 1994 book, The Billion-Dollar Molecule.^[3] His 2014 book, The Antidote: Inside the World of New Pharma, chronicled the company’s subsequent development over the next two decades.^[5]

By 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer.^[6]

In 2009, the company had about 1,800 employees, including 1,200 in the Boston area.^[2] By 2019 there were about 2,500 employees.^[7]

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.^[8] 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.^[9]

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.^[10] 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.^[11][12]

In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash.^[13] Semma Therapeutics created a «small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out.»^[14]

On 1 April 2020, former chief medical officer, Reshma Kewalramani, became president and chief executive officer of Vertex Pharmaceuticals.^[15] Former CEO and president Jeffrey Leiden transitioned to the role of executive chairman of the board of directors, as of 1 April 2020.^[15] Dr. Leiden will serve as executive chairman until April 2023.^[16][17]

In July 2022, the business announced it would acquire ViaCyte, Inc. for $320 million.^[18]

Medicines[edit]

Ivacaftor, lumacaftor and tezacaftor[edit]

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^[19] of the first drug, Kalydeco,^[20] 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 year.^[21][22]

Vertex also studied ivacaftor in combination with another drug (lumacaftor^[23]) 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.^[24][25]

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^[26] 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.^[27] 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.^[28]

On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis.^[29] On 12 February 2018, the FDA approved the combination, marketed as Symdeko.^[30]

Triple combination treatments[edit]

In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor.^[30] In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population.^[31]
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 new drug application was filed.^[32] Trikafta is approved for approximately 90% of patients with cystic fibrosis.^[33]

Vertex has refused to make Trikafta available in developing countries and works to block generic alternatives, making it inaccessible for thousands of patients.^[34]

Genetic therapies[edit]

• In 2015, Vertex entered into a research collaboration with CRISPR Therapeutics to develop gene-editing therapies for genetic diseases.^[35] 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.^[36]
• 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.^[37]
• In 2019, Vertex established a research collaboration with a privately held company Arbor Biotechnologies^[38] to discover novel proteins to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases.^[39] With seven different areas of focus in the deal, Vertex is paying up to $1.2 billion in milestones along with undisclosed up-front payments.^[38]
• Vertex’s product pipeline includes drug regimens targeting specialty diseases like sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, alpha-1 antitrypsin deficiency, pain, and APOL1-mediated kidney diseases.^[40]

Historical[edit]

Telaprevir[edit]

In 2014, Vertex discontinued telaprevir.^[41] 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.^[42]

CEO compensation[edit]

In 2015, several media outlets reported CEO Jeffrey Leiden’s 2014 compensation to be approximately US$48.5 million.^[43] Vertex shareholders opted for a reduction in CEO compensation in 2015 and 2016, resulting in 2016 earnings of US$17.4 million.^[44]

Pricing in the UK[edit]

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.^{[45][46][47][48][49]}

In March 2019, Vertex was legally required to destroy 7,880 packs of Orkambi that reached their expiry dates during negotiations with the NHS concerning the drug’s pricing.^[50][51]

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.^[52][33] The NHS in Wales and Northern Ireland will be offered equivalent pricing terms.^[53] On June 30, 2020, Vertex and NHS expanded the agreement to include reimbursement of Kaftrio, making CF patients in England to be among the first in Europe to access public funding for this medicine upon its approval by the European Commission.^[54]

By October 2019, Vertex’s CF medicines were publicly funded in 17 countries.^[55]

Venture philanthropy[edit]

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.^[56] 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.^[56][57] 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.^[56]

By 2015 the annual price of Kalydeco had been increased to more than $300,000 per patient.^[58]

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.»^[59]

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.^[60]

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.»^[59]

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.^[61] 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.»^[62]

See also[edit]

• Cystic Fibrosis Foundation
• Telaprevir

References[edit]

• Annual Report. Vertex Pharmaceuticals. 15 March 2004. Archived from the original on 3 March 2016. 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.

External links[edit]

• Official website
• Business data for Vertex Pharmaceuticals:
  • Google
  • SEC filings
  • Yahoo!

From Wikipedia, the free encyclopedia

Vertex Pharmaceuticals Incorporated

Type	Public company
Traded as	Nasdaq: VRTX Nasdaq-100 component S&P 500 component
Industry	Pharmaceuticals & Biotherapeutics[1]
Founded	1989; 34 years ago
Headquarters	Boston, Massachusetts, U.S.
Key people	Reshma Kewalramani, M.D., president and CEO Stuart A. Arbuckle, executive vice president and chief commercial officer Michael J. Parini, executive vice president and chief legal, administrative and business development officer Jeffrey Leiden, M.D., PhD, executive chairman David Altshuler, M.D., PhD, executive vice president, global research and chief scientific officer Amit K. Sachdev, executive vice president and chief patient officer
Products	Pharmaceuticals
Revenue	US$8.93 billion (2022)
Number of employees	4,500
Website	www.vrtx.com

Vertex Pharmaceuticals 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, Oxfordshire, England.

History[edit]

Vertex was founded in 1989 by Joshua Boger^[2] and Kevin J. Kinsella^[3] to «transform the way serious diseases are treated.»^[4]

The company’s beginnings were profiled by Barry Werth in the 1994 book, The Billion-Dollar Molecule.^[3] His 2014 book, The Antidote: Inside the World of New Pharma, chronicled the company’s subsequent development over the next two decades.^[5]

By 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer.^[6]

In 2009, the company had about 1,800 employees, including 1,200 in the Boston area.^[2] By 2019 there were about 2,500 employees.^[7]

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.^[8] 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.^[9]

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.^[10] 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.^[11][12]

In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash.^[13] Semma Therapeutics created a «small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out.»^[14]

On 1 April 2020, former chief medical officer, Reshma Kewalramani, became president and chief executive officer of Vertex Pharmaceuticals.^[15] Former CEO and president Jeffrey Leiden transitioned to the role of executive chairman of the board of directors, as of 1 April 2020.^[15] Dr. Leiden will serve as executive chairman until April 2023.^[16][17]

In July 2022, the business announced it would acquire ViaCyte, Inc. for $320 million.^[18]

Medicines[edit]

Ivacaftor, lumacaftor and tezacaftor[edit]

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^[19] of the first drug, Kalydeco,^[20] 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 year.^[21][22]

Vertex also studied ivacaftor in combination with another drug (lumacaftor^[23]) 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.^[24][25]

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^[26] 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.^[27] 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.^[28]

On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis.^[29] On 12 February 2018, the FDA approved the combination, marketed as Symdeko.^[30]

Triple combination treatments[edit]

In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor.^[30] In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population.^[31]
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 new drug application was filed.^[32] Trikafta is approved for approximately 90% of patients with cystic fibrosis.^[33]

Vertex has refused to make Trikafta available in developing countries and works to block generic alternatives, making it inaccessible for thousands of patients.^[34]

Genetic therapies[edit]

• In 2015, Vertex entered into a research collaboration with CRISPR Therapeutics to develop gene-editing therapies for genetic diseases.^[35] 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.^[36]
• 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.^[37]
• In 2019, Vertex established a research collaboration with a privately held company Arbor Biotechnologies^[38] to discover novel proteins to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases.^[39] With seven different areas of focus in the deal, Vertex is paying up to $1.2 billion in milestones along with undisclosed up-front payments.^[38]
• Vertex’s product pipeline includes drug regimens targeting specialty diseases like sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, alpha-1 antitrypsin deficiency, pain, and APOL1-mediated kidney diseases.^[40]

Historical[edit]

Telaprevir[edit]

In 2014, Vertex discontinued telaprevir.^[41] 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.^[42]

CEO compensation[edit]

In 2015, several media outlets reported CEO Jeffrey Leiden’s 2014 compensation to be approximately US$48.5 million.^[43] Vertex shareholders opted for a reduction in CEO compensation in 2015 and 2016, resulting in 2016 earnings of US$17.4 million.^[44]

Pricing in the UK[edit]

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.^{[45][46][47][48][49]}

In March 2019, Vertex was legally required to destroy 7,880 packs of Orkambi that reached their expiry dates during negotiations with the NHS concerning the drug’s pricing.^[50][51]

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.^[52][33] The NHS in Wales and Northern Ireland will be offered equivalent pricing terms.^[53] On June 30, 2020, Vertex and NHS expanded the agreement to include reimbursement of Kaftrio, making CF patients in England to be among the first in Europe to access public funding for this medicine upon its approval by the European Commission.^[54]

By October 2019, Vertex’s CF medicines were publicly funded in 17 countries.^[55]

Venture philanthropy[edit]

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.^[56] 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.^[56][57] 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.^[56]

By 2015 the annual price of Kalydeco had been increased to more than $300,000 per patient.^[58]

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.»^[59]

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.^[60]

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.»^[59]

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.^[61] 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.»^[62]

See also[edit]

• Cystic Fibrosis Foundation
• Telaprevir

References[edit]

• Annual Report. Vertex Pharmaceuticals. 15 March 2004. Archived from the original on 3 March 2016. 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.

External links[edit]

• Official website
• Business data for Vertex Pharmaceuticals:
  • Google
  • SEC filings
  • Yahoo!

Компания «ВЕРТЕКС»

«ВЕРТЕКС» – российская фармацевтическая компания из Петербурга, зарегистрирована в 1999 году, запуск первого производства состоялся в 2003 году.

В апреле 2020 года включена в перечни системообразующих предприятий Петербурга и России. Их формируют правительство Санкт-Петербурга и Министерство промышленности и торговли РФ.

По итогам 2019 года «ВЕРТЕКС» занимает 3 место среди российских производителей косметических средств и средств для ухода за полостью рта по доле продаж в аптеках РФ.

• 280+ позиций продукции в портфеле – лекарственных препаратов, косметических средств, биологически активных добавок;
• ~220 позиций лекарств. Это широкие линейки препаратов для применения в кардиологии, неврологии, гинекологии, оториноларингологии, аллергологии, дерматологии, гинекологии и др.;
• 4 оригинальных комбинированных лекарственных препарата, не имеющих аналогов. Они применяются в гинекологии, дерматологии, оториноларингологии и кардиологии;
• 65+ тысяч аптек с продукцией компании в России, а также Казахстане и Беларуси.

Инновационно-производственный комплекс

Фармкомплекс компании в особой экономической зоне «Санкт-Петербург» на участке «Новоорловская» входит в число крупнейших промышленных объектов и инвестиционных проектов ОЭЗ.

Его площадь превышает 56 тысяч м². Первая очередь была открыта в 2015 году, вторая и третья введены в эксплуатацию в 2019 году. Возможности площадки обеспечивают полный цикл от разработки до упаковки лекарств в различных формах выпуска: таблетках, капсулах, мазях, спреях, кремах и др.

Потенциальная мощность завода — более 200 миллионов упаковок продукции в год.

Признание

«ВЕРТЕКС» регулярно занимает ведущие позиции в рейтингах, становится обладателем престижных наград и премий, свидетельств общественного и профессионального признания. Компания и наши продукты постоянно входят в рейтинги максимальных значений и роста. Достижения отмечают региональные и федеральные органы власти.

• 2 место в рейтинге российских производителей лекарств, которые оказали наибольшее влияние на развитие фармрынка РФ (2018 и 2019 гг., Ipsos).
• Лауреат Премии Правительства РФ в области качества 2017 г. Среди 10 лауреатов — единственный фармпроизводитель, а также лидер по критерию «Процессы, продукция и услуги». Награду вручил Дмитрий Медведев в Доме Правительства РФ. О награждении лауреатов на сайте правительства РФ: http://government.ru/news/30068/.
• Дважды обладатель премий правительства Петербурга «Лучший инновационный продукт» в сфере медицины, биотехнологий и фармацевтики за оригинальные комбинированные лекарственные препараты: 2017 г. — для применения в гинекологии и дерматовенерологии; 2019 г. — для применения в дерматологии; третье место.
• Минпромторг РФ и губернатор Петербурга объявили благодарность компании за большой вклад в развитие фармацевтической промышленности в 2017 г.

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Вертекс

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