Oppenheimer analyst, Christopher Marai, recently published an analysis of the BioMarin (NASDAQ: BMRN) Kyndrisa briefing-documents and believes that Tuesday's panel discussions will end with hints of accelerated approval. Notes from his analysis follow:

They anticipate 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).

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, they 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.

The analyst believes "We're in new regulatory territory with greater focus on patients' needs, unmet need and regulatory flexibility. FDA has previously noted they are putting further emphasis on meaningful clinical outcomes and trends for rare diseases/unmet needs vs. statistical benefit on endpoints."

Dr. McDonald is the leading expert on 6MWT endpoints for DMD. He's long noted to the analyst clinical benefit from Kyndrsa in patients supporting use in DMD. In addition to a significant positive for panel, Dr. McDonald is seen as a positive for PTC Therapeutics (NASDAQ: PTCT).

FDA presentations are likely to be a rough spot. Dr. Ashutosh Rao's review is likely to include lackluster dystrophin production data. The statistical review is unlikely to instill confidence but the FDA has previously noted orphan indications' lack of statistically-significant benefit is not necessarily a deal killer.

The analyst anticipates patient discussion to focus on the urgent unmet need which they believe will lead to hints of accelerated approval.

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Shares of BioMarin Pharmaceutical Inc. closed at $97.00 yesterday.