Promising Parkinson’s therapy gets FDA investigational nod
June 06, 2024
The FDA designation will "create much greater momentum for potential cell therapies," says UCI Health Parkinson's expert Dr. Claire Henchcliffe.
Photo by Michael Neveux
Orange, Calif. — The promising investigational cell therapy bemdaneprocel for the treatment of Parkinson’s disease has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA).
RMAT-designated therapies are eligible for expedited review, guidance and a path to accelerated approval.
UCI Medical Center was the only academic medical center on the West Coast, and one of three in North America, to participate in the phase 1 trial designed by BlueRock Therapeutics to determine the safety profile of its investigational therapy, which aims to regenerate dopamine-producing neurons in the brain.
“We are excited about the positive data from the bemdaneprocel phase 1 clinical trial and believe it has great potential to help patients living with Parkinson's disease regain functions they have lost to the disease,” Seth Ettenberg, president and CEO of BlueRock Therapeutics LP, said in a statement announcing the FDA designation.
In March, UCI Health neurologist Claire Henchcliffe, MD, presented data at the Alzheimer’s and Parkinson’s Diseases Conference showng that the dopamine-producing neural cell therapy remained safe and well tolerated by participants in the phase 1 clinical trial at the 18-month mark.
“The RMAT designation for bemdaneprocel is outstanding news,” said Henchcliffe, chair of the UCI School of Medicine’s Department of Neurology and a principal investigator of the phase 1 trial.
“It will help efficiently advance the next stages of testing for this investigational cell therapy. More than that, I think this will create much greater momentum for potential cell therapies for Parkinson’s disease, bringing hope for better treatments.”
Bemdaneprocel is the most clinically advanced investigational cell therapy in the United States for treating Parkinson’s disease, according to BlueRock Therapeutics, a subsidiary of Bayer AG.
In addition to the phase 1 clinical trial data announced in March, researchers observed a continued increase in F-DOPA PET imaging signal after participants’ immune suppression therapy was halted at 12 months, demonstrating that the transplanted cells survived and engrafted in the brain.
The RMAT program is managed by the FDA’s Center for Biologics, Evaluation and Research (CBER) to review investigational regenerative medicines, including cell therapies. An RMAT designation makes bemdaneprocel eligible for expedited development review and development planning guidance from senior CBER officials.
According to BlueRock, it also “creates a pathway for early discussions about potential surrogate endpoints and ways to support accelerated approval and satisfy post approval requirements.”
Ettenberg said he looks forward to close collaboration with the FDA to prepare for phase 2 clinical studies of bemdaneprocel, which may begin later this year.
“We’re very excited for UCI Health to be a strong part of this program, especially with the prospect of a phase 2 clinical trial to begin soon that is focused on rigorously examining the clinical effects as well as collecting more safety data,” Henchcliffe said.
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UCI Health is the clinical enterprise of the University of California, Irvine, and the only academic health system based in Orange County. UCI Health is comprised of its main campus, UCI Medical Center, a 459-bed, acute care hospital in in Orange, Calif., four hospitals and affiliated physicians of the UCI Health Community Network in Orange and Los Angeles counties and ambulatory care centers across the region. Listed among America’s Best Hospitals by U.S. News & World Report for 23 consecutive years, UCI Medical Center provides tertiary and quaternary care and is home to Orange County’s only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center, gold level 1 geriatric emergency department and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us on Facebook, Instagram, LinkedIn and Twitter.
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