FDA approved EAP manufacturing at the Catalent Texas site

03 January 2022 | Monday | News

Three additional doses of NurOwn® will be made available to participants who completed the Expanded Access Protocol
Image Source : Public Domain

Image Source : Public Domain

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced plans for a dosing extension of NurOwn® for participants who completed the Expanded Access Protocol (EAP). The U.S. Food and Drug Administration (FDA) recommended that BrainStorm submit an EAP protocol amendment to provide additional dosing for these participants. Under the original EAP protocol, participants who had completed the Phase 3 NurOwn trial and who met specific eligibility criteria had the opportunity to receive 3 doses of NurOwn. Under the amended EAP protocol, these eligible participants will receive up to 3 additional doses. Data collected from the original EAP treatments informed the decision to move forward with additional doses for participants who completed it.

"This dosing extension for the expanded access protocol is an appropriate next step following the new analysis and biomarkers results of the Phase 3 study. It is deeply appreciated by our ALS patients. Eligible patients now have the opportunity to receive as many as 9 doses of NurOwn in total, allowing additional data collection to better understand the potential benefits of longer-term treatment," said Robert Brown, MD DPhil, Leo P. and Theresa M. LaChance Chair in Medical Research, and Chair, Department of Neurology, University of Massachusetts Medical School and UMass Memorial Medical Center, and one of the Principal Investigators in the NurOwn® Phase 3 study.

"I applaud BrainStorm for having the conviction to continue their EAP which has brought true hope to those in it. I am also grateful that they recognize the value and importance of EAPs and are willing to invest in doing them right despite being a small company. I look forward to the day, hopefully very soon, when every person living with ALS has access to NurOwn," stated Brian Wallach, person living with ALS, and Co-Founder of I AM ALS.

Chaim Lebovits, Chief Executive Officer, Brainstorm Cell Therapeutics, commented, "We are pleased to be able to provide additional treatments to these patients. This program is an outcome of a fruitful collaboration between the FDA, Patient Advocacy groups and Brainstorm. We look forward to continuing this dialogue with the FDA for the best path forward." 

The NurOwn EAP was established in 2020, following conclusion of the Phase 3 trial, and the protocol was developed in partnership with the FDA. Patients were treated at one of six clinical centers of excellence that participated in the Phase 3 trial. These six centers are: University of California, IrvineCedars-Sinai Medical CenterCalifornia Pacific Medical CenterMassachusetts General HospitalUniversity of Massachusetts Medical School; and Mayo Clinic. The FDA approved BrainStorm to manufacture NurOwn for the EAP Program at the Catalent facility in Houston, Texas.

According to the FDA, EAPs, alternatively known as "compassionate use" programs, provide a pathway for appropriate patients to receive an investigational medicine for treatment of a serious disease or condition outside of a clinical trial when no comparable or satisfactory alternative therapy options are available.

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