Just as BioMarin thought it was nearing the FDA finish line for its hemophilia A gene therapy Roctavian, the agency has moved back its decision date by three months.
After BioMarin recently submitted new three-year data on its drug, the agency has decided it needs more time to review the application.
Roctavian's new decision date at the agency is June 30, BioMarin said in a release. The delay comes after BioMarin submitted a "substantial amount" of additional data to support its application, the company said.
"We are continuing to work closely with FDA and appreciate the agency's active engagement as we seek to deliver this important therapy to patients with severe hemophilia A," BioMarin’s president of R&D, Hank Fuchs, M.D., said in a statement.
The delay comes even as BioMarin prepares for the U.S. launch. That work includes hashing out a warranty contract for payers and mapping out a plan for treatment centers around the country, executives said on a recent conference call.
Roctavian has traveled a long road to the FDA finish line. Before its initial rejection, the gene therapy scored a breakthrough therapy designation way back in 2017.
But in 2020, BioMarin received a complete response letter from the FDA, with the agency voicing concerns over the duration of the drug's benefit. BioMarin resubmitted its latest application last September.
This time around, the FDA has already completed a pre-license inspection of the manufacturing facility that produces Roctavian. The company has estimated 2023 revenues for Roctavian will come in between $100 million and $200 million.