Final ICER evidence report on SCD gene therapies exa-cel and lovo-cel

The USA’s health technology assessor, the Institute for Clinical and Economic Review (ICER), today released a Final Evidence Report assessing the comparative clinical effectiveness of two sickle cell disease gene therapies.

This examined the value of exagamglogene autotemcel (exa-cel), from Vertex Pharmaceuticals (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP), and lovotibeglogene autotemcel (lovo-cel), from bluebird bio (Nasdaq: BLUE), for sickle cell disease (SCD).

For adolescents and adults with severe SCD who do not have access to, or cannot receive, hematopoietic stem cell transplantation (HSCT) from a matched sibling or haploidentical donor, a majority of panelists (13-1) found that current evidence is adequate to demonstrate a net health benefit for exa-cel when compared to standard of care (ie, hydroxyurea, chronic blood transfusions, pain medication, iron chelation).