Cystic Fibrosis: Global Clinical Trial Landscape (2024)

Cystic Fibrosis (CF), a genetic disorder stemming from CFTR gene mutations, affects chloride and bicarbonate transport, resulting in thick mucus in organs like the lungs and pancreas. Recent advancements have extended life expectancy, with a projected 75% increase in adult CF population by 2025.

Treatment focuses on comprehensive management, including nutritional support, airway clearance techniques, and targeted drugs like CFTR modulators, which enhance lung function by addressing the defective CFTR protein. However, treatment effectiveness varies based on gene mutations.

Globally, CF affects 162,400 individuals, with significant prevalence among the White population. While prevalent in regions like North America and Europe, CF is also emerging in regions like the Middle East and Asia. Personalized approaches and robust healthcare initiatives are crucial for addressing global CF challenges.

Since 2018, over 450 CF trials have been initiated, with Europe and North America leading. Vertex Pharmaceuticals Inc. is conducting Phase III trials for small molecule therapies targeting the CFTR protein, reflecting ongoing efforts to enhance CF treatment.

By downloading the report below, readers will gain valuable insights on patient enrollment, site selection, and navigating global regulatory landscapes tailored to rare diseases, thus propelling forward CF research and treatment.