An experimental cell therapy being developed by Capricor Therapeutics appeared to preserve heart and muscle function in young patients with Duchenne muscular dystrophy, in a late-stage trial published in a top-ranked medical journal on Wednesday.
But a panel convened by the U.S. Food and Drug Administration voted 9-3 against recommending approval, citing insufficient evidence of the treatment's efficacy and concerns about changes to how the company measured the outcomes.
Approval of the drug would represent a potentially important advance for the condition that primarily affects boys.
The data marks the first time a treatment has been shown to slow or halt not only the loss of upper limb function, but progression of heart failure in boys and young men with Duchenne, researchers said. Duchenne muscular dystrophy is an inherited disease that progressively weakens muscles throughout the body, with heart failure often leading to early death.
The disease is caused by a mutation on the X chromosome.
The treatment, deramiocel, is manufactured from cells taken from healthy hearts donated for transplantation but not usable for that purpose.
In the late-stage trial, 106 participants ages 10 to 22 with advanced disease received either deramiocel or a placebo intravenously every three months for one year.
The therapy slowed weakening of skeletal muscles by 54% and slowed heart dysfunction by 91%, the researchers reported in The Lancet.
“if young people maintain their upper limb function, it will help preserve their ability to do normal activities, such as feeding themselves. The heart benefits could reduce mortality,” study coauthor Dr. Craig McDonald of UC Davis Health said in a statement.
Some patients even experienced a slight improvement in heart function.
The researchers had previously found that infusions of so-called cardiosphere-derived cells regenerated damaged heart muscle after heart attacks. Later, they found these cells secrete packets of RNA molecules that help repair the body’s tissues.
The researchers said they plan to continue studying the therapeutic effects of deramiocel, including whether it can be used to treat other diseases.
Study leader Dr. Eduardo Marbán of Cedars-Sinai Health Sciences University, whose wife is Capricor's CEO, said the Phase 3 study was the first "to show that a cell therapy is effective against a genetic disease, or any type of heart disease.”
The FDA is expected to issue its decision by August 22.
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