Investigational PD Cell Therapy Receives FDA RMAT Designation

The US Food and Drug Administration (FDA) granted the investigational cell therapy bemdaneprocel a Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of Parkinson disease (PD), announced bemdaneprocel developer BlueRock Therapeutics, a subsidiary of Bayer AG. The designation qualifies the cell therapy for expedited development review and development planning guidance from senior managers at the FDA Center for Biologics, Evaluation and Research. It also creates a pathway for early discussions about potential surrogate end points and ways to support accelerated approval and post approval requirements.

Bemdaneprocel cells are designed to replace dopamine-producing neurons in people with PD. During surgery, neuron precursors derived from human embryonic pluripotent stem cells are implanted into the brain. Afterward, they have the potential to reform neural networks that have been severely affected by PD and to restore motor and nonmotor function to patients, according to the developer.

The RMAT designation follows phase 1 trial results announced in March that showed bemdaneprocel was well tolerated with no major safety issues over 18 months. The open-label phase 1 study included 12 patients with PD who underwent surgical transplantation of bemdaneprocel cells and a year-long immunosuppression regimen.

Trial results also showed an increase in the F-DOPA positron emission tomographic imaging signal after patients stopped immune suppression therapy at 12 months. The finding indicates that the transplanted cells survive and engraft in the brain, BlueRock Therapeutics reported. “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 PD regain functions they have lost to the disease,” said Seth Ettenberg, president and CEO of BlueRock Therapeutics. “Now with this RMAT designation in hand, we look forward to closely collaborating with the FDA to ready this program for phase II clinical studies.”

Reference

BlueRock Therapeutics receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate bemdaneprocel. News release. BlueRock Therapeutics; May 30, 2024. Accessed June 3, 2024. https://www.bluerocktx.com/bluerock-therapeutics-receives-fda-regenerative-medicine-advanced-therapy-designation-for-parkinsons-disease-cell-therapy-candidate-bemdaneprocel/