FDA Extends Review of Capricor’s DMD Cell Therapy by 3 Months

The U.S. Food and Drug Administration (FDA) has extended its review of Capricor Therapeutics’ candidate treatment for Duchenne muscular dystrophy (DMD).

According to Fierce Biotech on the 25th (local time), the FDA delayed its decision while evaluating additional information submitted by Capricor after a negative advisory committee vote.

The FDA had originally been expected to rule on the application by Aug. 22. But expectations for approval waned after the advisory committee voted against the cell therapy’s efficacy, with 9 votes against and 3 in favor.

Capricor partly revived expectations earlier this month by submitting additional data and announcing plans to shift the application’s focus to a different endpoint.

The FDA agreed to review the additional data and extended the review period by 3 months. Capricor will now be notified by Nov. 22 whether its cell therapy candidate, deramiocel, is approved.

Even so, investors value Capricor less than they did before the negative advisory committee vote. This suggests that doubts about the likelihood of approval remain.

Capricor shifted the application’s focus from cardiomyopathy, the subject of the negative vote, to upper limb function. The advisory committee did not vote on whether deramiocel improves upper limb function, so it did not reach a conclusion on the new indication.

Capricor CEO Linda Marbán said earlier this month during an earnings call that the advisory committee’s discussion of upper limb function was “directionally supportive of the clinical evidence.”

However, several experts questioned the evidence related to upper limb function. One attendee noted, “It is very difficult to say there is a real effect in terms of strength.”

Capricor’s revised application includes clinical data that the advisory committee had not seen. Over the next 3 months, the FDA will assess whether the additional 1 year of follow-up data provided by Capricor, together with the existing evidence, supports approval.

The FDA raised concerns about the existing evidence in a briefing for the advisory committee in July. The agency said Capricor’s phase 3 clinical trial did not demonstrate a statistically significant or clinically meaningful effect on the prespecified upper limb function endpoint.

Capricor presented statistically significant analysis results for the primary endpoint, but regulators concluded that the results were difficult to interpret.