Vertex Readies For Commercial Future For Multi-Therapeutic Area R&D Strategy

Vertex Pharmaceuticals Incorporated has been executing well on an R&D strategy to expand the portfolio beyond the company’s core therapeutic area of cystic fibrosis and now, driven by that success, Vertex will soon need to prove it can execute commercially across multiple therapeutic areas as well.

The company could be launching products in various new areas, including sickle cell disease (SCD), beta-thalassemia, pain and more in the near term. Vertex filed for approval in Europe and in the UK for exagamglogene autotemcel (exa-cel), an autologous, ex vivo CRISPR/Cas9 gene-edited therapy, for SCD and transfusion-dependent beta-thalassemia (TDT). A rolling biologics licensing application (BLA) with the US Food and Drug Administration is on track for completion by the end of the first quarter