Following the news that the UK Medicines and Healthcare Products Regulatory Agency has granted conditional marketing authorisation to Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, marking the world’s first approval of a CRISPR-based gene editing therapy; Joselia Carlos, Medical Device Analyst at GlobalData, offers her view: “The approval of Casgevy for sickle cell anemia (SCA) and β-thalassaemia heralds a new era where the potential to cure genetic disorders becomes a tangible reality, offering hope to millions worldwide grappling with inherited conditions.
“The US FDA is currently considering approval of Casgevy for SCA. The global cell and gene therapy market is expected to reach $80 billion in 2029, with a high compound annual growth rate of 51.6 per cent between 2023 and 2029, according to GlobalData forecasts. With clustered regularly interspaced short palindromic repeats (CRISPR) officially making its long-awaited debut in the therapeutic space, Vertex Pharmaceuticals will likely gain a dominant position in the global cell and gene therapy market.
“The limiting factor for widespread usage of this drug is its high cost. Estimates suggest that it could cost approximately $2 million per patient. To reduce Casgevy’s costs and ensure its safety and effectiveness, ongoing research, ethical oversight, and collaboration among scientists, policymakers, and healthcare providers are imperative.”