The labeling update limits the “therapy’s indication to ambulatory patients,” the FDA said. The labeling must no longer contain an indication that the treatment is for non-ambulatory patients with DMD.
Furthermore, 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.
In both cases, the patients ended up getting hospitalized two months after they received Elevidys treatment.
Following the safety communication, Sarepta voluntarily ceased the distribution of Elevidys for use in ambulatory patients, the FDA said.
In addition to the fatal cases, 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.
Revised Guidelines, Company Response
Sarepta’s Boxed Warning describes the risk of “serious liver injury and acute liver failure, including fatal outcomes.”The labeling should update the Warnings and Precautions, Adverse Reactions, Dosage and Administration, Clinical Studies, and other relevant sections, the FDA said. It must also include a new Medication Guide for patients and caregivers.
According to the FDA, the revised labeling should recommend that users conduct weekly liver function tests for at least three months after treatments, with the individuals advised to remain near a medical facility for at least two months following the therapy.
The labeling must warn patients who experience yellowing of the skin or eyes, or a change in mental status, to immediately seek medical attention. It should also contain a warning that Elevidys is not recommended for patients with preexisting liver impairments, recent or active infections, or those who have received recent vaccinations.
“The FDA is requiring the manufacturer to conduct a postmarketing observational study to further assess the risk of serious liver injury,” the agency said.
“The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.”
To date, the gene therapy has been administered to more than 1,100 individuals worldwide in both clinical and real-world settings, it said.
“We want to thank the FDA for their thorough and collaborative review. Completion of the safety labeling change for ELEVIDYS will ensure that families and healthcare professionals have clear information, supported by a Medication Guide, to help understand these updates and guide treatment decisions,” said Louise Rodino-Klapac, president of research & development and technical operations at Sarepta.
On July 28, the FDA informed Sarepta that it recommended continuing shipments of the therapy for ambulatory patients. The suspension for non-ambulatory patients remained.
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