FDA grants RMAT designation to SanBio’s cell therapy

The US Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to SanBio Group’s cell therapy, SB623, to treat chronic neurological motor deficits secondary to traumatic brain injury (TBI).

SB623 is an investigational cell therapy using modified and cultured mesenchymal stem cells from adult bone marrow after temporary genetic modification.

Implantation of these cells into injured nerve tissue in the brain allows restoration of lost motor functions by stimulating the organ’s natural regenerative ability.

RMAT granted designation due to SB623’s clinical data, including the Phase II STEMTRA trial conducted over 12 months to assess the safety and efficacy of the cell therapy.

During the Phase II study, researchers compared SB623 with surgery in 61 patients with stable chronic neurological motor deficits secondary to TBI at 13 surgical and 18 assessment sites across the US, Japan and Ukraine.

Researchers implanted the cells around the brain injury site. The primary endpoint was mean change from baseline in Fugl-Meyer Motor Scale (FMMS) score, a measure of motor impairment changes at six months.

SB623 met the primary endpoint, demonstrating an average of 8.3 point improvement in the FMMS from baseline, compared to 2.3 in the control group, at 24 weeks.

Of all participants who received cell therapy, 18 achieved a ten or more point FMMS improvement versus one control.

The study did not reveal any new safety signals and the most common adverse effect was headaches.

SanBio chief medical officer and research head Bijan Nejadnik said: "The RMAT designation for SB623 is an important regulatory milestone for SanBio as we investigate it as a treatment option for patients with chronic neurological motor deficits resulting from a traumatic brain injury.

"TBIs are one of the most common health conditions worldwide that often cause long-term complications or death. We look forward to working with the FDA on a potentially accelerated clinical development programme to address this serious unmet medical need."

The therapy also secured the Sakigake designation from the Japanese Ministry of Health, Labour and Welfare.

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