This sustained them between their three breakthroughs, from HIV to Hepatitis C and finally having the CF projects to fall back on when Incivek fell out from under them. It seemed that Leiden’s strategy worked out for Vertex, saving them from going under just a few years earlier.During the 2000s Vertex had mostly focused on getting Incivek onto the market; however, they still had a team working away on Cystic Fibrosis projects that were acquired 10 years earlier with the Aurora Biosciences acquisition. Vertex to get new investment from Cystic Fibrosis Foundation under revised deal – The Boston Globe. Retrieved April 8, 2018, from https://www.marketwatch.com/story/vertex-sees-higher-expenses-stock-dropsOver the next decade Vertex ran into problems with many of their projects failing to make it through to clinical trials, however, in 2011 they finally saw a second successful drug make it through approval. 96 Issue 7 | Chemical & Engineering News. Rather than looking at the rare disease as challenging, he saw the advantages that Vertex had over the competition when developing drugs for this disease. Reinvesting some of their revenue into drugs outside of the Cystic Fibrosis category will allow them to be diverse in drug discovery, and could have the potential of producing another breakthrough.Vertex Pharmaceuticals originated as a small pharmaceutical start-up company that was founded in 1989 by Josh Boger. As a small molecule, VX-765 could be taken orally and was thus thought to have excellent market potential. Vertex Pharma’s 3-drug combo aids breathing in cystic fibrosis patients. He had an illustrious academic career, starting from elementary school (where he skipped a grade), to high school (which he left early after being accepted to university during his junior year).
Using this unique model they wanted to develop breakthrough drugs that tackled difficult diseases where there weren’t current treatment options. Cystic Fibrosis is considered a rare disorder with only approximately 75 000 people (in USA, Europe and Australia) diagnosed with it (“Understanding CF | Vertex Pharmaceuticals,” n.d.). While it may seem that this condition should be simplistic, with only a single gene being implicated in CF, it turns out that there are many (over 2000) mutations that have been identified in humans, with 127 of these directly linked to causing CF, thus adding to the complexity of treating CF. These conditions were so severe that Jillian and her family were considering a lung transplant to help alleviate these symptoms, something that would be difficult to obtain, due to a shortage of donors and the cost to her family. It also claimed to target the cause of the disease rather than just treating the symptoms, which is what, up until now, Jillian had been receiving during her long hospital visits. Vertex Pharmaceuticals and the Cystic Fibrosis Foundation: Venture Philanthropy Funding for Biotech.
Retrieved April 8, 2018, from http://www.boston.com/business/gallery/inside_vertex/Staton, T. (2013). There is a lot of controversy over Leiden setting this high price point for the drug.
http://www.iconplc.com/icon-files/docs/Rare-Disease-Brochure.pdfWhile it was always understood that CF was genetically linked and spread through heredity, it wasn’t until 1989 the one specific gene was identified, named the Cystic Fibrosis Transmembrane Regulator (CFTR) Gene. While there are many things that lead to them to the company they are today, it seems that there are three key factors continued to their current success. Biomed Pharmacother 2007;61:191–3. The company then went public in 1991, to raise funds (making around $25 million dollars). Vertex Pharmaceuticals (Germany) GmbH mit Sitz in München ist im Handelsregister mit der Rechtsform Gesellschaft mit beschränkter Haftung eingetragen. Das Unternehmen wird beim Amtsgericht 80333 München unter der Handelsregister-Nummer HRB 192987 geführt. This was not an easy task for Vertex. Their method was unique as they heavily invested in research and development into the diseases they wanted to develop drugs for and they specifically focused on using new techniques that utilized computer software to identify potential drug targets base on molecule structure. This strategy is in line with their “all in for CF” strategy that Leiden brought to the table. This is because the drug only worked for a small percentage of the CF patients (around 3000) and because only a small percentage are affected by the mutation that Kalydeco deals with.
This research into the genetics and on the CFTR gene (which had been ongoing for the past 12 years) paid off as the team had found a molecule that offered CF treatment that was targeting the actual protein malfunction due to mutation. This is especially true when considering the risk that many drug trials fail, with only 13.8% of drugs making it from initial discovery through to approval (Cross, 2018). One can imagine that these are only a temporary solution and CF patients needed a drug that could treat the cause.There are also attempts from Vertex to improve their public relation which include them starting their own CF programs to give back to the community from their profits. Retrieved April 1, 2018, from https://www.bostonglobe.com/magazine/2017/12/13/vertex/zO9My0GXMQ4rjYz28CbIhI/story.htmlHistoric Stock Lookup | Vertex Pharmaceuticals. Vertex vermarktet den HIV-Proteasehemmer Lexiva gemeinsam mit GlaxoSmithKline. A disorder is considered rare when less than 200 000 people are affected by it (ICON, 2018).