The U.S. Food and Drug Administration (FDA) has announced that the agency is now recommending the removal of Sarepta Therapeutics’ voluntary hold for ambulatory patients eligible to receive ELEVIDYS. In its own press release, Sarepta reported that it will resume shipping ELEVIDYS to sites of care for treatment of ambulatory patients with Duchenne imminently. This update comes after Sarepta announced last week that the company voluntarily and temporarily paused all shipments of ELEVIDYS for Duchenne in the United States, regardless of ambulatory status.
According to the FDA, its investigation concluded that the death of the 8-year-old boy in Brazil in June was unrelated to treatment with ELEVIDYS and confirmed that Sarepta can resume shipments. Both Sarepta and the FDA indicated that they will continue to collaborate to complete the safety label update for ELEVIDYS and to discuss an approach to risk-mitigation for non-ambulatory patients, who remain subject to a voluntary, temporary pause.
According to Sarepta, the company will be reaching out to sites of care that had infusions scheduled for July and August to inform them of the availability of ELEVIDYS for ambulatory patients. For families, your clinical team will be available to answer questions, help you understand how this may impact you or your child’s care, and offer guidance on making decisions about treatments and care going forward.
PPMD deeply appreciates the FDA’s acknowledgment of the Duchenne community’s voice and its ongoing commitment to listening and engaging with those most impacted by this disease. We remain dedicated to working with the agency to ensure that the needs, hopes, and lived experiences of individuals and families affected by Duchenne continue to inform every step of the decision-making process.
Read Sarepta’s community letter here.
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