The FDA has extended its review of Capricor Therapeutics’ Duchenne muscular dystrophy (DMD) drug candidate, deferring the decision while the agency assesses information the biotech submitted after a negative advisory committee vote.
The FDA was originally set to rule on the filing by Aug. 22, although investors’ hopes of approval had deflated after an advisory committee voted nine to three against the efficacy of the cell therapy. Capricor partly reinflated those hopes earlier this month by outlining plans to submit extra data and refocus its filing on a different endpoint.
The FDA agreed to review the extra data, triggering a three-month extension. Capricor is now set to learn if the agency will approve its cell therapy candidate, deramiocel, by Nov. 22.
Even so, investors continue to ascribe less value to Capricor than before the negative advisory committee vote, suggesting doubts about its chances of winning approval. The advisory committee discussion and briefing document the FDA prepared (PDF) for the meeting contain statements that support skeptical interpretations of Capricor’s chances.
Capricor has shifted its submission from cardiomyopathy—the subject of the negative vote—to upper limb function. Because the advisory committee never voted on whether deramiocel improves upper limb function, the meeting delivered an inconclusive verdict on Capricor’s new indication.
The advisory committee’s discussion of deramiocel’s effect on upper limb function “was directionally supportive of the clinical evidence,” Capricor CEO Linda Marbán, Ph.D., said on the company’s earnings call earlier this month. Yet multiple experts questioned the evidence on upper limb function, with one attendee saying that “it’s very hard to say that there is a real effect in terms of muscle strength.”
Capricor’s revised submission contains clinical data that were unavailable to the advisory committee. Over the next three months, the FDA will assess whether the extra year of follow-up provided by Capricor, plus the original evidence, support approval.
The agency raised concerns about the original evidence in its July briefing of the advisory committee. Capricor’s phase 3 trial failed to show a statistically significant or clinically meaningful effect on the pre-specified upper limb function endpoint, the FDA said. The biotech presented a statistically significant analysis of the primary endpoint, but the regulator concluded the results were hard to interpret.
Facts Only
* The FDA extended the review of the DMD drug candidate, deramiocel.
* The extension was to assess information submitted after a negative advisory committee vote.
* The original review deadline was August 22.
* Capricor outlined plans to submit extra data and refocus its filing on a different endpoint.
* The FDA agreed to review the extra data, triggering a three-month extension.
* The new decision date for approval is November 22.
* The advisory committee voted nine to three against the efficacy of the cell therapy.
* Capricor shifted its submission from cardiomyopathy to upper limb function.
* A subset of experts questioned the evidence regarding muscle strength effects on upper limb function.
* The FDA found the Phase 3 trial failed to show a statistically significant or clinically meaningful effect on the pre-specified upper limb function endpoint.