July 13, 2024

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FDA Approves Expansion of DMD Gene Therapy Elevidys for Patients Over 4 Years Old

FDA Approves Expansion of DMD Gene Therapy Elevidys for Patients Over 4 Years Old



FDA Approves Expansion of DMD Gene Therapy Elevidys for Patients Over 4 Years Old

On June 20, the U.S. Food and Drug Administration (FDA) approved Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) for an expanded indication. The therapy is now approved for use in patients aged 4 and older with Duchenne muscular dystrophy (DMD), regardless of ambulatory status, and who carry a DMD gene mutation.

The FDA’s decision includes full approval for all patients aged 4 and older diagnosed with a DMD gene mutation, while non-ambulatory patients received accelerated approval. The latter approval is contingent on the results of an ongoing Phase III study.

FDA Approves Expansion of DMD Gene Therapy Elevidys for Patients Over 4 Years Old

In their decision, the FDA noted that evidence provided by Sarepta demonstrated clinical benefit. Although the original study did not meet its primary endpoint, the review of secondary and exploratory endpoints was “compelling” enough to confirm clinical benefit.

Last June, the FDA granted accelerated approval for Elevidys for DMD gene mutation children aged 4 and 5 who were still ambulatory. Despite the limited number of patients initially treated, this global first gene therapy for DMD offered new hope for the future.

On June 12, Pfizer’s DMD gene therapy fordadistrogene movaparvovec faced a setback when its Phase III CIFFREO clinical trial failed to meet its primary endpoint: the study did not show improved motor function in treated boys aged 4 to 7 compared to placebo.

The approval of Elevidys for a broader patient population is undoubtedly a significant benefit for those with DMD. It also raises hopes for further breakthroughs in DMD gene therapy research.

FDA Approves Expansion of DMD Gene Therapy Elevidys for Patients Over 4 Years Old

(source:internet, reference only)


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