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Biomarin Pharmaceutical Inc (NASDAQ:BMRN) announced Wednesday that the U.S. Food and Drug Administration (FDA) informed them that they no longer plan to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN.
The news resulted in Biomarin shares jumping 8% and saw analysts at Stifel and Truist react positively.
“The review of a BLA is a dynamic process, and we appreciate FDA’s ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A,” said Hank Fuchs, President of Worldwide Research and Development at BioMarin. “We look forward to further dialogue with the Agency as it reviews our application.”
Stifel said the news is “probably a positive” and reiterated a Buy rating on BioMarin.
“We believe yesterday’s QURE/CSL hemeB HEMGENIX approval holds favorable read-throughs onto the gene therapy space and, specifically for BMRN/Roctavain, on FDA’s comfort with approving GTx for a disease has other good therapeutic options. That said, there are two distinctions with BMRN’s submission – durability and questions around steroid use – that, given the FDA’s original communicated plans for an AdComm, indicated that Roctavian presents different and more nuanced review issues. Conversely, news today that FDA now no longer plans to hold a panel for Roctavian (note: there also wasn’t a panel for HEMGENIX) appears to be a positive,” the analysts wrote.
Truist analysts also reiterated a Buy rating on the stock following the news, telling investors in a note that it is an “extreme positive.”
“We view this as very positive for BMRN and for the pending approval of Roctavian,” wrote the analysts. “We remain bullish on the approval of Roctavian and remind investors that BMRN is on track to host the FDA’s inspection of their gene therapy manufacturing facility, which we believe would not be happening if the FDA did not intend to approve the drug.”
