The drug manufacturer has been commanded to include and highlight the adverse health impacts on the label.
By Catholics for Catholics
After two deaths of two children, the Food and Drug Administration approved an updated safety warning for a gene therapy treatment that restricts its use, the agency said in a statement on Nov. 14.
Sarepta Therapeutics Inc, the pharmaceutical company granted the approval for Elevidys, a one-time gene therapy treatment for Duchenne muscular dystrophy (DMD) b. DMD makes muscle cells vulnerable to injury, leading to gradual muscle degeneration, according to a story by The Epoch Times.
Elevidys is a prescription gene therapy used to medicate ambulatory and non-ambulatory individuals aged at least 4 years old who suffer from DMD and have a confirmed mutation in the dystrophin gene. Ambulatory means they are patients who can walk and are not restricted to bed.
According to The Times, the labeling update limits the “therapy’s indication to ambulatory patients,” the FDA said. The labeling must no longer have a warning that the treatment is for non-ambulatory patients with DMD.
In addition, the new labeling includes a Boxed Warning—the agency’s “most prominent” safety warning.
“These actions follow reports of fatal acute liver failure in non-ambulatory patients treated with the product,” the FDA said.
The FDA said in a June 24 safety communication that it was probing the death cases.
The two patients who died were male and had high levels of transaminase, which are enzymes released by the liver into the bloodstream. High levels of transaminases indicate that the liver is under stress.
The patients ended up getting hospitalized two months after they received Elevidys treatment. Following the safety communication, Sarepta voluntarily stopped the distribution of Elevidys for use in ambulatory patients, the FDA said.
Besides the cases that ended in deaths, there has also been a non-fatal case of acute liver injury, with the individual suffering complications such as vein thrombosis, necrosis, and bowel ischemia, it said.
“After a comprehensive evaluation of the available safety data, FDA has now approved substantial labeling revisions for Elevidys,” the agency said.
Sarepta’s Boxed Warning describes the risk of “serious liver injury and acute liver failure, including fatal outcomes.”
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