By Zachary Stieber
Contributing Writer
Sarepta Therapeutics will not agree to a regulatory request to pause shipments of a treatment for muscular dystrophy in the wake of patient deaths, the pharmaceutical company said on Friday after receiving the request.
“Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population,” Sarepta said in a statement.
Two patients have died after receiving Elevidys, a gene therapy for Duchenne muscular dystrophy. A third died after receiving a different gene therapy, also produced by Sarepta, for limb girdle muscular dystrophy, the company said on Friday.
Food and Drug Administration officials then said they were investigating the deaths and that they had asked Sarepta to voluntarily and immediately halt shipments of Elevidys, but that the company refused.
Earlier this year, Sarepta stopped shipping Elevidys to non-ambulatory patients after reporting a second death in a patient who had received the treatment. Both of the patients who died were non-ambulatory, as was the patient who received the other therapy.
Sarepta maintains there are no safety concerns for ambulatory patients.
“Elevidys is the only approved gene therapy for individuals devastated by Duchenne, a rare, progressive and ultimately fatal disease,” the drugmaker said. “We are committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy.”
Duchenne muscular dystrophy is an inherited disorder with no cure that worsens over time, resulting in the decline of muscle function, but treatment can help patients.
The FDA did not return a request for comment. Officials said they have placed on hold Sarepta’s trials evaluating the therapy for limb girdle muscular dystrophy and revoked the platform technology designation for the company’s delivery platform, given safety concerns.
They are allowing Sarepta to continue selling Elevidys to ambulatory patients, though they said further action may be taken.
“Today, we’ve shown that this FDA takes swift action when patient safety is at risk.” FDA Commissioner Dr. Marty Makary said in a statement. “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”
Dr. Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research, said the agency “will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.”
