ICER finds BioMarin’s Hemophilia Gene Therapy Could Have Warranted a Record Price Tag

Hemophilia is a very expensive disease to treat. Therapies that provide replacement clotting protein are not entirely effective but still carry a hefty six-figure price tag. BioMarin Pharmaceutical’s gene therapy for hemophilia, coming in at US$2-3 million, was set to be the worlds most expensive drug before being rejected by the US Food and Drug Administration. However, a drug-price watchdog groups said that under certain conditions, the high-cost of the therapy could be cost effective for the healthcare system.

“Even a gene therapy that comes in at a couple million dollars, presuming durability, can be worthwhile financially speaking,” Michael Sherman, chief medical officer at Harvard Pilgrim, a New England insurer, said in a recent interview with BioPharma Dive. It will take approximately another year and a half for BioMarin to collect the data needed to resubmit Roctavian to the FDA for approval. Read more here.