The FDA granted supplemental approval for Casgevy (exagamglogene autotemcel) in patients aged 2 and older with sickle cell disease or transfusion-dependent beta thalassemia, expanding from the prior 12-and-older label. Casgevy is co-developed and co-commercialized by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP).
Who cashes in: Vertex Pharmaceuticals (VRTX) is the commercial lead on Casgevy and captures the larger revenue share; expanding the eligible population to younger children meaningfully increases the addressable patient pool for a therapy that already carries a list price above $2 million per treatment. CRISPR Therapeutics (CRSP) shares in royalties and milestone payments — the label expansion is a direct catalyst for both companies. Beam Therapeutics (BEAM) and Editas Medicine (EDIT) are coattail names: they are developing next-generation gene-editing therapies for the same disease indications, and FDA's willingness to approve and expand CRISPR-based treatments in pediatric populations validates the regulatory path for their own pipelines.