Statement on “Trickett Wendler, Frank Mongiello, Jorden McLinn, and Matthew Bellina Right to Try Act of 2017”
The ‘‘Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017’’ will enable terminally ill patients who do not meet clinical trial criteria to seek access to investigational therapies that have passed Phase1 trials, continue in active development and have not yet received marketing authorization by the United States Food and Drug Administration (FDA). When signed into law by President Trump, it will amend the Food, Drug and Cosmetic Act to create a new pathway for terminally ill patients to seek access to investigational therapy when they have exhausted approved treatment options and are unable to participate in a clinical trial.
BrainStorm understands the serious unmet medical need and urgency of people living with ALS and their families. We continue to receive countless inquiries regarding ALS patient access to NurOwn® through the Right to Try pathway. We are awaiting details of the new law and how it will be implemented by the Food and Drug Administration for us to draft a policy and how best to proceed. We have been, and will continue to be engaged and collaborate with people with ALS, their families and caregivers, ALS experts, legislators and government agencies to ensure that the all voices are heard in this important discussion. BrainStorm is currently assessing the Right to Try Act and its potential implications and when signed into law, will issue a formal policy on Right to Try in early June.
BrainStorm remains focused and fully committed to completing the Phase 3 clinical trial of NurOwn® as quickly as possible. Our first obligation to the ALS community is to complete the Phase 3 trial and as such our resources, including financial and manufacturing, are fully devoted to advancing the phase 3 clinical trial. We are continually evaluating opportunities to provide access to NurOwn® outside of the current Phase 3 trial to the many patients who can’t wait. However, the most expedient way to make NurOwn® available to all is by completing the necessary clinical trial data collection and endpoint analyses and submitting the required marketing applications without delay to regulatory agencies, including the FDA.
Enrollment continues for the BrainStorm multi-dose, randomized, double-blind, placebo-controlled Phase 3 trial at all 6 U.S. sites. For more information on enrollment in the NurOwn® Phase 3 trial contact one of the clinical trial sites listed below for details and screening: http://www.brainstorm-cell.com/patients-caregivers/clinical-trials/
Statement on Expanded Access/Compassionate Use
We are currently not offering compassionate use or expanded access to NurOwn®. BrainStorm understands the serious unmet medical need and the urgency of people living with ALS. We are dedicating all our resources to moving forward with the clinical development program for NurOwn® and initiating the phase 3 clinical trial that we hope will prove NurOwn® to be safe and effective for treating ALS. We believe completing the necessary clinical trial and obtaining the data needed for review and approval by the by regulatory agencies including the U.S. Food and Drug Administration (FDA) is the most expedient way to make NurOwn® available to the greatest number of patients.
The personalized medicine nature of NurOwn® stem cell therapy adds to the complexity for its provision under an expanded access/compassionate use program. The product is manufactured using a process that involves taking a sample of bone marrow from a patient and then sending the bone marrow to a manufacturing facility where Mesenchymal Stem Cells are isolated and grown under carefully managed conditions.
Hospital Exemption Program Update- Israel
We continue to explore options and advocate to support the hospital exemption program in Israel for a limited number of patients. Outside of Israel, we are engaged in regulatory level and health authority discussions. Please fill out this Form and we will provide updates on our progress as they become available.
If you have additional questions, feel free to reach out to us via our Contact page.
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