Montreal, May 14, 2020 – Biomarin announced on May 11 its gene therapy currently awaiting approval by the U.S. Food and Drug Administration will be called Roctavian.
A ruling on market approval in the U.S. for Roctavian, also called valoctocogene roxaparvovec, is expected on August 21.
If approved, the uptake of Roctavian could be slowed by the COVID-19 pandemic. Biomarin’s chief commercial officer, Jeffrey Ajer, said, “The biggest variable will be the willingness of certain hospitals to do infusions at the time of the launch. If we are so fortunate to have an approved Roctavian product in Q4, the launch will probably be somewhat dependent on the course of the COVID-19 pandemic.”
In February, Clarivate Analytics forecasted that, if approved, valoctocogene roxaparvovec could see more than $1 billion in annual sales by 2024. The report quoted the list price could be $2.5 to 3 million, which would make it the most expensive drug ever to reach the market. The company said in May that it is talking to payers about outcomes-based reimbursement agreements.
Biomarin has yet to announce an application to Health Canada for marketing approval.