FDA Adcomm Cancels Meeting on BioMarin's Hemophilia A Gene Therapy

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BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A.

BioMarin's stock jumped 7.05% Wednesday following the news. 

The BLA was resubmitted on Oct. 12. When BioMarin announced the FDA had accepted its resubmission, it stated the agency did not indicate there would be an advisory committee meeting. 

This changed when BioMarin released its third-quarter report on Oct. 26, in which the company stated the FDA did, in fact, plan to meet. Neither the FDA nor BioMarin specified a date for the meeting.

In a conference call following that third-quarter report, Jeffrey Ajer, BioMarin's EVP and CMO, said his team was preparing for launch based on the target action date of Mar. 31, 2023.

This news comes one day after another gene therapy for hemophilia made headlines. On Tuesday, the FDA approved CSL Behring and uniQure’s Hemgenix (etranacogene dezaparvovec-drlb), a one-time gene therapy developed for adults with Hemophilia B. 

At $3.5 million, Hemgenix is now the most expensive medicine in the world. If approved, BioMarin’s Roctavian will likely have a similar price tag. 

As BioSpace previously reported, Roctavian is expected to be priced at $2.5 million. In the same conference call, Ajer said the Institute for Clinical and Economic Review (ICER), a drug-pricing watchdog in the U.S., issued a report that suggested the possible U.S. price was appropriate.

"At a presumed price of $2.5 million, the report referred to Roctavian as a dominant treatment relative to emicizumab on cost-effectiveness. Roctavian was projected to have lower costs of $4 million, slightly higher quality-adjusted life years and slightly lower bleeds," Ajer said.

Hemophilia A is an X-linked genetic disorder caused by missing or defective clotting protein Factor VIII. Hemophilia A patients often experience spontaneous bleeds into their muscles and joints. 

Roctavian is an adeno-associated virus serotype 5 (AAV5)-based gene therapy vector that expresses a recombinant version of human factor VIII. The therapy has already been approved in Europe. 

The BLA is based on multiple clinical trials studying Roctavian in severe hemophilia A, including GENEr8, a Phase III study, and an ongoing Phase I/II dose escalation study. 

In Wednesday’s announcement, BioMarin stated it still plans to host the previously-scheduled FDA Pre-Licensure Inspection (PLI) of its gene therapy manufacturing facility in Novato, California.

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