Written By: Pharmacally Medical News Desk
BlueRock Therapeutics’ investigational cell therapy Bemdaneprocel (BRT-DA01) has been granted the Pioneering Regenerative Medical Product designation (Sakigake) by Japan’s Ministry of Health, Labour and Welfare (MHLW), marking a key regulatory milestone as the therapy advances toward potential approval for Parkinson’s disease treatment. This recognition is meant to accelerate development and regulatory consultation in Japan, giving BlueRock a structured pathway for interaction with authorities as it works to bring this novel approach to patients.
Parkinson’s disease is a progressive neurodegenerative disorder affecting roughly 10 million people worldwide and about 250,000 in Japan, with symptoms that include tremors, rigidity, and loss of motor control. Current treatments focus on symptom relief rather than restoring lost nerve function.
What the Sakigake Designation Means
Japan’s Sakigake system supports groundbreaking therapies by providing prioritized regulatory consultations and a more efficient review process. It is similar in intent to the United States’ Regenerative Medicine Advanced Therapy (RMAT) designation and the EU’s advanced therapy frameworks, designed to help promising treatments reach patients sooner.
With this designation, Bemdaneprocel may benefit from more frequent dialogue with the MHLW on clinical development plans and, if the therapy meets criteria, could qualify for accelerated approval and priority review in Japan.
About Bemdaneprocel
Bemdaneprocel is an investigational cell therapy derived from human embryonic pluripotent stem cells. Designed to replace dopamine-producing neurons that are lost in Parkinson’s disease, the therapy uses dopaminergic neuron precursors that mature after surgical implantation into the brain. The goal is to rebuild damaged neural networks and potentially improve both motor and non-motor symptoms, moving beyond current treatments that mainly manage symptoms without addressing underlying neuronal loss.
In the United States, Bemdaneprocel has previously received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, which support accelerated development and review for promising regenerative treatments.
Clinical Progress and Future Development
BlueRock has advanced Bemdaneprocel into a pivotal Phase III clinical trial called exPDite-2, a randomized, sham surgery-controlled study that is enrolling participants to evaluate safety and efficacy in people living with Parkinson’s disease.
Earlier phase data showed favorable safety profiles and encouraging trends in motor function outcomes up to 36 months after treatment in Phase I studies, reinforcing the potential of cell replacement approaches in this disease area.
Gabi Belfort, MD, PhD, Bemdaneprocel Product Lead, BlueRock Therapeutics, said the Sakigake designation recognizes the urgent need for innovative treatments in Parkinson’s disease and will enable close collaboration with Japanese regulators to advance Bemdaneprocel efficiently for patients and their families.
Christian Rommel, PhD, Global Head of Research and Development, Bayer Pharmaceuticals, said the designation underscores Bemdaneprocel’s potential to transform Parkinson’s disease treatment and reflects Bayer’s commitment to working with regulators worldwide to improve patient access to innovative therapies.
References
BlueRock Therapeutics’ investigative cell therapy, bemdaneprocel, for treating Parkinson’s disease receives Pioneering Regenerative Medical Product designation in Japan, 17 December 2025, https://www.bayer.com/media/en-us/bluerock-therapeutics-investigative-cell-therapy-bemdaneprocel-for-treating-parkinsons-disease-receives-pioneering-regenerative-medical-product-designation-in-japan/
BlueRock Therapeutics’ investigative cell therapy Bemdaneprocel for treating Parkinson’s disease receives Pioneering Regenerative Medical Product designation in Japan, 17 December 2025, https://www.bluerocktx.com/bluerock-therapeutics-investigative-cell-therapy-bemdaneprocel-for-treating-parkinsons-disease-receives-pioneering-regenerative-medical-product-designation-in-japan/
BlueRock Therapeutics reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson’s disease, 07 October 2025, https://www.bluerocktx.com/bluerock-therapeutics-reports-positive-36-month-results-from-phase-i-trial-of-bemdaneprocel-for-treating-parkinsons-disease/