A panel of advisers to the regulator said on Tuesday Vertex Pharmaceuticals and CRISPR Therapeutics could assess potential safety risks of their sickle cell disease gene therapy after approval.
An influential US drug pricing watchdog raised the price estimate of two experimental gene therapies from Vertex Pharmaceuticals/CRISPR Therapeutics and bluebird bio for sickle cell disease.
Vertex Pharmaceuticals and CRISPR Therapeutics s one-dose gene editing therapy for sickle cell disease would be cost effective if priced at up to $1.9 million, an influential US drug pricing group said on Wednesday.