Oppenheimer On BioMarin Panel: 'We're Not In Kansas Anymore'

• Last Friday, the FDA released briefing documents for a meeting to discuss drisapersen (Kyndrisa) which will take place on Tuesday.
• The documents focused on efficacy concerns and dystrophin data. In a report issued Friday, Oppenheimer analysts explained that, “Documents suggest safety signals, while not a deal killer for accelerated approval, will need monitoring and hurt Kyndrisa in the market vs. competitive eteplirsen (especially with blackbox warning).” Thus, they assured they continued to envision an accelerated approval of Kyndrisa but are not as bullish with respect to the market.
• In another report issued Monday, the experts, Christopher Marai and Michelle Gilson, shared some thoughts after a more detailed analysis of the documents. This article will focus on these ideas.

Marai and Gilson analyzed the Kyndrisa briefing documents and decided to shift their focus to the discussions and outcome of Tuesday’s panel, based on the proposed itinerary.

The experts expect “content and expert commentary for the panel to start on a high note, followed by a difficult discussion on dystrophin and statistics, with a moderately positive patient session to conclude with a narrowly positive overall tone (and possible vote).” They anticipate the benefit risk clinical perspective summary will also have a positive tone.

“Briefing documents suggest that FDA could be amenable to accelerated approval based upon positive P2 study ('117) (p=0.014, 6MWT 35m-benefit at 25 weeks). However, we are concerned about potential expectation bias due to injection site reaction in this study, noting BMRN's analysis only addresses the 48-wk time point, not the P2 primary endpoint at 25 weeks,” they expounded.

“We're Not In Kansas Anymore—Pre-Panel Regulatory Expert Commentary

May Not Be Relevant.”

The FDA seems to be implementing FDASIA in DMD therapeutic review. This suggests that the regulatory landscape has changed, with focus shifting towards “patients' needs, unmet need and regulatory flexibility.” In fact, the FDA had noted in previous occasions that it is “putting further emphasis on meaningful clinical outcomes and trends for rare diseases/unmet needs vs. statistical benefit on endpoints.”

Disclosure: Javier Hasse holds no positions in any of the securities mentioned above.