What is Vertex Pharmaceuticals? It is a pharmaceutical company that manufactures more than 4 different drugs in the US. Its products are marketed under a variety of names in other countries, and its website does not list non-US versions of the drugs. However, Vertex does ask patients to rate the quality of its medicines based on the effectiveness of the treatment, as well as the ease with which they are taken.
During the last decade, the company has made big moves in the market. Incivek, a treatment for cystic fibrosis, reached $1.1 billion in sales in 2012. Its subsequent decline came as a result of a deal with Gilead Sciences, a company backed by billionaire Peter Thiel. In the meantime, the company has been steadily expanding, and it has recently acquired Semma Therapeutics, a company that aims to use stem cells to cure Type 1 diabetes.
The company is currently working on the next phase of development for a new treatment for cystic fibrosis. CTX001 is undergoing phase one clinical trials for patients with sickle cell disease and beta thalassemia. CTX001 is a departure from Vertex’s usual fare. While most of the company’s CF therapies are based on traditional pharmacology, CTX001 uses genetic editing technology to develop a drug to treat cystic fibrosis. Vertex has partnered with CRISPR Therapeutics for the development of the drug.
The company is expanding its leadership in cystic fibrosis with the approval of Trikafta, a revolutionary treatment for cystic fibrosis. In just five years, Vertex is aiming to reach 90% of patients with cystic fibrosis. It has already reached this goal with current treatments. Meanwhile, the company is planning to receive regulatory approvals for new CF treatments in the near future.