United States of America

FDA Advisory Committee Evaluates Muscular Dystrophy Treatment

Is Deramiocel a breakthrough for DMD patients or built on flawed trial data?

Published JUL 29

FDA Advisory Committee Evaluates Muscular Dystrophy Treatment

Story

Above: The U.S. Food and Drug Administration headquarters in Silver Spring, Maryland, on Apr. 28. Image credit: Valerie Plesch/Bloomberg/Getty Images

The Facts

The Food and Drug Administration's (FDA) Cellular, Tissue and Gene Therapies Advisory Committee met on Wednesday to review Capricor Therapeutics' Biologics License Application (BLA) for deramiocel, a cell therapy for cardiomyopathy in Duchenne muscular dystrophy.
U.S. Food and Drug Administration
BioPharm International
STAT
In December, Capricor announced that its HOPE-3 clinical study had met both its primary endpoint — a 54% slowing of upper-limb disease progression — and its key cardiac secondary endpoint, with p-values of 0.029 and 0.041, respectively, in a randomized, double-blind, placebo-controlled trial involving 106 participants.
Capricor Therapeutics
The FDA, however, released briefing materials ahead of Wednesday's committee meeting, casting doubt on Capricor's findings, which it stated showed "no statistically significant difference between deramiocel and placebo at 12 months."
U.S. Food and Drug Administration
Despite this, on the day of the review, The Lancet published Capricor's HOPE-3 study, which the company subsequently described as validating its results due to the medical journal's "rigorous, independent peer review process."
The Lancet
Capricor Therapeutics
Members of the committee will vote on whether the evidence presented at the meeting demonstrates the treatment's effectiveness. The vote, however, will only table a non-binding recommendation, with final approval resting with the FDA.
U.S. Food and Drug Administration
U.S. Food and Drug Administration
The meeting is part of a two-day review of medicines and therapies the agency has previously disregarded, with the committee examining the merits of a BLA for Replimune's vusolimogene oderparepvec, which has been rejected twice, on Thursday.
Politico
U.S. Food and Drug Administration

Sources Split

LEFT

CENTER

RIGHT

ESTAB
CRIT

CENTER

PRO
ESTAB

The Spin

Narrative A

The FDA's own briefing makes clear that Capricor's HOPE-3 trial failed to meet its pre-specified primary and secondary endpoints, and that the company then altered its statistical analysis plan after unblinding, a move that undermines the entire evidentiary foundation. Approving a therapy on this record would set a dangerous precedent for rare disease drug development.

U.S. Food and Drug Administration

The Clinical Trial Vanguard

Narrative B

Capricor's HOPE-3 study definitively shows that it met its primary skeletal muscle endpoint and key cardiac endpoint, with deramiocel slowing upper-limb decline by 54% and preserving heart function. The publication of these results in The Lancet, with its rigorous independent peer review, is telling and further validates the trial's design, methodology and findings.

Capricor Therapeutics