ICER suggests $1.9M price tag for pending sickle cell disease drugs
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The influential drug pricing watchdog, Institute for Clinical and Economic Review (ICER) says that upcoming sickle cell disease therapies, exa-cel, and lovo-cel, would be cost-effective if priced at up to $1.9M.
"From the earliest days of gene therapy, patients, families, and clinicians have imagined that someday it might be possible to address the underlying genetics of sickle cell to achieve a cure," ICER's Chief Medical Officer, David Rind, said, disclosing the findings in a draft report Wednesday.
ICER's views are released as developers of exa-cel and lovo-cel, Vertex Pharmaceuticals (NASDAQ:VRTX)/ CRISPR Therapeutics (NASDAQ:CRSP), and bluebird bio (NASDAQ:BLUE), are targeting a potential FDA approval of the treatments, also known as exagamglogene autotemcel and lovotibeglogene autotemcel, respectively.
"These first two genetic therapies, using different technologies and altering different genetic targets, may mean that day has nearly arrived," Rind added.
CRISPR (CRSP) and Vertex (VRTX) recently completed the rolling submission of their marketing application for exa-cel, an autologous CRISPR/Cas9 gene-edited therapy.

Bluebird bio (BLUE) plans to seek the FDA's priority review for gene therapy lovo-cel in a marketing application expected to be submitted this year before the planned launch in 2024.