Washington, D.C. The U.S. Food & Drug Administration said on Friday that it has opened an inquiry into the death of an 8-year-old child who was treated with Elevidys, a gene therapy for Duchenne muscular dystrophy created by Sarepta Therapeutics.
The FDA stated that the agency has formally requested and obtained a voluntary suspension of Elevidys distribution in the United States while safety concerns are assessed in light of the child’s June 7 death, who received treatment in Brazil.
Elevidys is an intravenous (IV) gene therapy administered once. According to the Muscular Dystrophy Association, it is intended to assist kids with Duchenne muscular dystrophy, a rare and dangerous genetic disorder that gradually weakens muscles. According to the FDA, the treatment delivers a copy of a protein called micro-dystrophin, which helps promote muscle strength in males who lack a functional form of the gene that makes it, using a harmless virus.
Despite the fact that the youngster did not participate in a clinical trial, post-marketing surveillance has recorded his death. According to Roche, the company that sells Elevidys outside of the United States, the treating physician determined that the death had nothing to do with the medication. The FDA stated that it is still cautious in spite of this.
This is the third recent death linked to Sarepta’s gene therapy, according to Reuters: Following treatment, two teenage boys reportedly passed away from severe liver failure, and a 51-year-old man who was getting SRP 9004, an experimental treatment that used the same viral vector, also passed away.
Sarepta initially opposed the FDA’s order to stop distribution in the United States. However, the firm changed its mind and decided to halt Elevidy’s shipments in response to increasing regulatory pressure and a precipitous drop in its stock price, according to the Associated Press. The suspension can be in effect for a few months.
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