Vertex shares plans ahead of exa-cel regulatory decision

Nov. 7, 2023

Vertex Pharmaceuticals unveiled its exa-cel approval plans and 2024 projections in its Q3 2023 earnings report.

The company reported robust sales growth for its cystic fibrosis (CF) products, projecting $9.85 billion in revenue for 2023. This growth, Vertex claimed, was driven by Trikafta's success and an expanded CF product portfolio.

Ahead of significant regulatory approvals, which include its collaboration with CRISPR Therapeutics, Vertex has halted the development of VX-864, a first-generation AATD corrector for Duchenne muscular dystrophy (DMD), citing non-serious rash events in certain patients. 

CRISPR Therapeutics and Vertex are jointly developing gene-edited treatments targeting beta-thalassemia and sickle cell disease. Last week, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee held its meeting for exa-cel, designed to treat severe sickle cell disease (SCD) in individuals aged 12 and older experiencing recurrent vaso-occlusive crises.

On the earnings call, Vertex CEO Reshma Kewalramani referred to exa-cel as "a large commercial opportunity" sharing that the recent AdComm meeting "represented a significant milestone for Vertex."

The drug is the first therapeutic product resulting from the 2015 collaboration between the companies. If approved, exa-cel would become the first gene edited therapy and functional one time cure for the approximately 20,000 severe SCD patients in the U.S. The FDA has given exa-cel priority review status for SCD treatment, with a PDUFA action date set for December 8, 2023. Additionally, the BLA for exa-cel in transfusion-dependent beta-thalassemia is scheduled for PDUFA review on March 30, 2024. 

Kewalramani noted that Vertex recently submitted a marketing authorization application for exa-cel to the Saudi Food and Drug Authority, SFDA, and that exa-cel was the first medicine ever to receive Breakthrough designation by the SFDA.