FDA Not Changing Etep Stance (SRPT) (BMRN) - Leerink
The updated FDA briefing documents ahead of Sarepta's (NASDAQ: SRPT) eteplirsen AdCom (4/25) remain largely negative citing concerns with trial design, efficacy, dystrophin measurement methods, and laboratory measurements and statistical analysis. This version of the documents also includes the FDA's rebuttal of the addendum to its briefing materials that SRPT submitted in January, stating that “we do not agree with the applicant’s characterization of inaccuracies in the initial FDA briefing document.”
Leerink analyst, Joseph Schwartz, has been cautious on the name and maintained his Market Perform rating and $13 PT on SRPT. The analyst expects a similar outcome to the BioMarin (NASDAQ: BMRN) advisory panel which was also negative.
The analyst noted that the updated FDA review is even more negative on the historical control data. 4-yr. clinical effectiveness data submitted in January at the time was considered as major amendment to eteplirsen NDA that had resulted in the 3-mos. delay in PDUFA action date. Having had multiple months to review this additional submission, the FDA has become even more critical by identifying many potential confounding factors, including two matched historical control patients that were reported to have lost ambulation but also had 10m walk test values reported as well as a secondary analysis showing that when 6MWT scores are plotted based on pt age (rather than years of treatment) that there is substantial overlap between eteplirsen-treated patients and the control group.
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Shares of Sarepta Therapeutic closed at $19.71 yesterday.