RESNA Welcomes Three Members to Board of Directors
The Rehabilitation Engineering and Assistive Technology Society of North America (RESNA) has announced that three newly elected board members will begin their terms on Aug. 1.
In a July 12 social media post, RESNA welcomed Ana Allegretti, Ph.D., OTR, ATP; Julienne Brown, PT, DPT, NCS, ATP/SMS; and Cara Masselink, Ph.D., OTR/L, ATP, HCPS.
The new board members will serve their three-year terms through July 31, 2028.
Allegretti is an associate professor at the University of Texas Health Science Center at San Antonio. Brown is a physical therapist at the assistive technology department/outpatient wheelchair clinic at Bryn Mawr Rehabilitation Hospital. Masselink is an associate professor at Western Michigan University College of Health and Human Services.
FDA Halts Sarepta Therapeutics Limb-Girdle MD Clinical Trials, Asks for Duchenne MD Drug Shipments to Be Paused
The U.S. Food & Drug Administration (FDA) said it has halted Sarepta Therapeutics’ (Nasdaq: SRPT) investigational gene therapy clinical trials for limb-girdle muscular dystrophy “following three deaths potentially related to these products and new safety concerns that the study participants are or would be exposed to an unreasonable and significant risk of illness or injury,” the agency said in a July 18 press release.
The FDA said it also revoked Sarepta Therapeutics’ platform technology designation. The company declined to voluntarily stop its shipments of Elevidys, used to treat Duchenne muscular dystrophy, after being asked to do so by the FDA on July 18.
All three deaths are believed to have been caused by acute liver failure, the FDA said. One of the deaths was of a Sarepta Therapeutics trial participant with limb-girdle muscular dystrophy, while the other two deaths were of patients with Duchenne muscular dystrophy treated with Elevidys.
“Today, we’ve shown that this FDA takes swift action when patient safety is at risk,” FDA Commissioner Marty Makary, M.D., M.P.H., said in the press release. “We believe in access to drugs for unmet medical needs, but are not afraid to take immediate action when a serious safety signal emerges.”
Sarepta Therapeutics said in a July 18 press release that it “received an informal request” from the FDA the afternoon of July 18 to pull Elevidys from the market. “We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports,” Sarepta Therapeutics said. “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. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care.”
The company noted it had already paused shipments of Elevidys for non-ambulatory Duchenne patients “while we work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure.” Fierce Pharma reported that one patient treated with Elevidys died in March, and a second Elevidys patient died in June. Both Duchenne patients who died were non-ambulatory.
Sarepta Therapeutics said Elevidys was not the gene therapy used in the now-halted limb-girdle clinical trials.
“Elevidys is the only approved gene therapy for individuals devastated by Duchenne, a rare, progressive and ultimately fatal disease,” Sarepta Therapeutics 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.”
