Capricor Therapeutics’ share value dropped sharply after the Food and Drug Administration’s advisory committee voted 9-3 against recommending approval of its cell therapy, deramiocel, for Duchenne muscular dystrophy cardiomyopathy, according to a July 31 interview with BioSpace. The meeting was contentious, and Chief Executive Officer Linda Marbán said she is not ruling out taking the matter to court.
When asked about possible legal action, Marbán said, “I don’t know.” She continued, “I like to collaborate. I’m a consensus builder. My number one goal is to meet with the agency and figure out a path forward that works for everybody. If they really dig their heels in and they say there’s no path forward for this therapeutic, we’ll explore strategic options for the therapy. Pull it off of the Duchenne space, and yeah, pursue legal action.” The FDA did not respond when asked if it was concerned about potential litigation.
The advisory committee’s vote followed an analysis focused on statistical analysis plan (SAP) 1.1—a draft Capricor said was never intended as part of its approval application but used by the FDA as a benchmark because it was in place at study initiation. During her presentation at the meeting, FDA biostatistical analyst Tingting Zhou referenced SAP 1.1 as foundational for evaluating trial data.
Marbán argued that using SAP 1.1 skewed results unfavorably: “It was very clear from the voting question and sort of how [FDA] pitched it with stat plan 1.1 that they seem to have some type of ulterior motive in trying to take this therapeutic down, and I can’t figure out why,” she said Thursday. She added that other versions of Capricor's SAP showed statistical significance supporting deramiocel's efficacy.
Patient advocate Mindy Leffler also raised concerns about how data were analyzed during Wednesday’s meeting: “There were some really disingenuous things that were done to put together the analysis that they came up with,” Leffler said after testifying alongside her son Aidan Leffler, who participated in an earlier trial phase.
With several weeks remaining before deramiocel’s Aug. 22 action date at the FDA, Marbán indicated Capricor will continue discussions with regulators or may seek opportunities outside the United States if necessary: “I think there’s some appetite on my board to go outside the U.S., go to Europe and finish the work there and get approval there,” she said.
