Vertex Pharmaceuticals is a leading biopharmaceutical company focused on developing transformative medicines for individuals with serious and life-threatening diseases. Their mission is to improve patient outcomes through the development of innovative therapies. Vertex conducts rigorous clinical trials to evaluate the safety and efficacy of their investigational medicines.

These trials provide the foundation for obtaining regulatory approvals, which are necessary for widespread patient access to their medicines. The company is committed to raising awareness about clinical trials and encourages patient participation. Clinical trials offer individuals the opportunity to access investigational medicines before regulatory approval, providing potential therapeutic options for those in need.

In cases where participation in a clinical trial is not possible and the patient's medical condition is critical, Vertex may consider special access to their investigational medicines. This access, known as early access or compassionate use, is granted on a case-by-case basis and in accordance with applicable laws and regulations. Vertex's approach to early access prioritizes patient safety and comprehensive evaluation of individual requests.

It requires objective clinical criteria, unmet medical need, and sufficient data supporting a benefit-risk assessment. The company is dedicated to ensuring sustainable access to their medicines by pursuing regulatory approval and establishing pathways for all eligible patients. Vertex Pharmaceuticals is committed to transforming patients' lives through groundbreaking therapies and continuous scientific innovation