- BioMarin Pharmaceutical Inc. BMRN shares have crashed over the past five trading days, falling 11.54 percent to a low of $95.17 on November 20.
- Ying Huang of Bank of America Merrill Lynch has maintained a Buy rating on the company, with a price objective of $165.
- With the share price having declined to the levels seen prior to the Prosensa acquisition, and with the risk-reward skewed to upside, Huang remains a buyer of the stock.
Analyst Ting Huang mentioned that for drisapersen, BioMarin Pharma “sees clinically meaningful benefit in the Phase 2 studies, FDA views the difference in 6MWD between continuous and intermittent drisapersen cohorts as inconsistent.”
Although the panel did not overtly agree with the FDA, the voting suggests that the efficacy of the drug was inconclusive.
Related Link: Oppenheimer On Upcoming BioMarin Panel: Efficacy Concerns Circulate
Huang mentioned that dystrophin continues to be a “controversial biomarker,” a majority of the panel expressing a belief that the lack of meaningful dystrophin data “not integral in interpreting results, given that there is currently no data showing a strong correlation between dystrophin levels and clinical benefit.”
The FDA also concluded that the current means of quantifying dystrophin are unreliable. “Given the absence of clarity around the biomarker, we do not believe the dystrophin levels will play an important role in FDA decision making,” Huang stated.
Regarding the safety of drisapersen, the FDA focused on adverse events associated with the drug, while patient advocate groups and the panelists believe, “almost unanimously,” that “patients and their family should have the power to decide if they are comfortable with the safety profile as long there is efficacy.”
For now, the outcome of the PDUFA, scheduled for December 27, remains unclear.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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