The FDA, essentially, told SRPT to fuck off. It’s looking rather ominous for the prospects for their muscular dystrophy drug.
This Advisory Committee meeting was initially scheduled to take place on January 22, 2016, but had to be rescheduled because of a weather emergency. Since the initial FDA briefing materials were released, the applicant submitted additional information about clinical outcomes of patients in Study 201/202, and also made public an addendum to their briefing materials in which the applicant describes what it calls “key inaccuracies” in the briefing document FDA released in advance of the original date for this Advisory Committee meeting. As will be discussed below, and in more detail in the Cross-Disciplinary Team Leader summary document, we do not agree with the applicant’s characterization of inaccuracies in the initial FDA briefing document.”
“… patients in Study 201/202, and also made public an addendum to their briefing materials in which the applicant describes what it calls “key inaccuracies” in the briefing document FDA released in advance of the original date for this Advisory Committee meeting. As will be discussed below, and in more detail in the Cross-Disciplinary Team Leader summary document, we do not agree with the applicant’s characterization of inaccuracies in the initial FDA briefing document…”
“Based on the data submitted by the applicant, considerable doubt remains about how much, or perhaps even whether, dystrophin levels were increased by eteplirsen. The degree of uncertainty about the dystrophin data hinders discussion of its use as surrogate endpoint for eteplirsen. However, to the degree that the dystrophin data may be interpretable, the amount and distribution of dystrophin in treated patients appears to be within the range typically associated with DMD, not BMD. Data suggesting that higher levels of dystrophin were produced by eteplirsen appear unreliable.”
Piper Jaffray believes this equates to a likely denial by the FDA.
SRPT is absolutely plunging today.
XON too