VERTEX PHARMACEUTICALS INC / MA
Vertex Pharmaceuticals Incorporated is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. The company's business strategy is to discover and develop innovative medicines by combining transformative advances in the understanding of human disease and the science of therapeutics to dramatically advance human health. Vertex's business segments include cystic fibrosis (CF), sickle cell disease and beta thalassemia (SCD/TDT), pain, APOL1-mediated kidney disease, type 1 diabetes, myotonic dystrophy type 1, and alpha-1 antitrypsin deficiency. The company's website is <https://www.vrtx.com>, and its ticker symbol is VRTX. Vertex's marketed products for CF include TRIKAFTA/KAFTRIO, SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO. These medicines are used by nearly three quarters of the approximately 92,000 people with CF in North America, Europe, and Australia. In SCD and TDT, Vertex's therapy, CASGEVY, was recently approved in the US, EU, UK, Saudi Arabia, and Bahrain for the treatment of severe SCD and TDT. The company is also developing new therapies for these diseases, including a triple combination of vanzacaftor/tezacaftor/deutivacaftor for CF and myeloablative conditioning agents and small molecules for SCD and TDT. In pain, Vertex is developing NaV1.8 inhibitors, including VX-548, which has shown positive results in Phase 3 clinical trials for the treatment of moderate-to-severe acute pain following abdominoplasty or bunionectomy surgery. The company also has a next-generation NaV1.8 inhibitor, VX-993, in development. In addition, Vertex is evaluating multiple small molecules for the potential treatment of APOL1-mediated kidney disease, and is developing non-autologous, fully differentiated, stem-cell derived islet cell therapies for type 1 diabetes. The company is also collaborating with Entrada on an oligonucleotide-based approach for myotonic dystrophy type 1 and is investigating small molecule correctors for alpha-1 antitrypsin deficiency. Vertex's commercial organization focuses on supporting the appropriate use of its medicines in the markets where they have been approved, and its sales and marketing organizations are responsible for promoting products to healthcare providers, ensuring effective distribution, ensuring safe use, and obtaining reimbursement from third-party payors. The company markets its products through personal interactions with physicians and allied health care professionals, and its government affairs and public policy group advocates for policies that promote life sciences innovation and increase awareness of the diseases on which Vertex is focusing.