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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.
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Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta (US) and Kaftrio (EU), 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.
Reshma Kewalramani, is the president and chief executive officer of Vertex Pharmaceuticals, a biotechnology company based in Boston, Massachusetts, as of April 1, 2020. She is the first female CEO of a large US biotech company. She was previously the chief medical officer and vice president of global medicines development and medical affairs at Vertex.
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 the Senior Vice President and Site Head of the San Diego Research Center of 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.
References
- 1 2 3 4 5 "The 21st Annual Wiley Prize in Biomedical Sciences". Events & Lectures. Retrieved 2023-09-27.
- ↑ "DuPont To Acquire CombiChem For $95 Million". www.pharmaceuticalonline.com. Retrieved 2023-09-27.
- ↑ "Heroes of Chemistry - Archive". American Chemical Society. Retrieved 2023-09-27.
- ↑ "Breakthrough Prize – Life Sciences Breakthrough Prize Laureates – Sabine Hadida". breakthroughprize.org. Retrieved 2023-09-27.
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