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ALS community calls upon the Food and Drug Administration to reevaluate BrainStorm's NurOwn, citing recent survival data

BrainStorm Cell Therapeutics endorses a Citizens' Petition lodged at the FDA, urging the organization to sanction its therapy product, NurOwn. Following the withdrawal of its Biologics License Application (BLA) in 2023, the company had planned to initiate a Phase IIIb trial this past month.

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ALS advocacy group urges FDA to reexamine BrainStorm's NurOwn treatment, highlighting recently...
ALS advocacy group urges FDA to reexamine BrainStorm's NurOwn treatment, highlighting recently disclosed survival data.

ALS community calls upon the Food and Drug Administration to reevaluate BrainStorm's NurOwn, citing recent survival data

In a recent development, BrainStorm Cell Therapeutics, a biotech company, has found itself at the centre of a significant decision by the Food and Drug Administration (FDA) following a Citizens' Petition requesting a reconsideration of NurOwn's approval for Amyotrophic Lateral Sclerosis (ALS).

## Background of the Petition

The petition, submitted by ALS patients and advocates, seeks to present new data supporting NurOwn's efficacy. This data includes survival data, improvements in biomarkers such as neurofilament light (NfL), and functional outcomes in early-stage ALS patients.

## New Evidence Highlighted

The petition emphasises statistically significant changes in 23 CSF biomarkers, including NfL, which indicate reduced neurodegeneration. Subgroup analyses also show that NurOwn recipients retained more points on the functional scale compared to placebo groups.

## FDA's Consideration and BrainStorm's Position

The FDA is currently considering the petition, offering an opportunity for a fresh review of NurOwn's data. BrainStorm Cell Therapeutics supports the petition and welcomes the FDA's willingness to reassess the data. The company remains committed to advancing access to NurOwn for ALS patients and plans to proceed with a Phase 3b clinical trial.

## Ongoing Clinical Trials

BrainStorm is moving forward with the Phase 3b ENDURANCE trial, designed to enroll 200 early-stage ALS patients. The trial aims to validate NurOwn's efficacy through a randomised, double-blind, placebo-controlled phase followed by an open-label extension.

The outcome of the FDA's decision will be closely watched, particularly under Commissioner Marty Makary's emphasis on "common sense" approvals for life-threatening diseases. The decision will depend on how the FDA weighs the new evidence against its stringent approval criteria.

This story has been updated to include further details from BrainStorm on the Phase IIIb trial of NurOwn. The Phase IIIb trial was initially scheduled to begin last month. BrainStorm withdrew the Biologics License Application (BLA) prior to its PDUFA date and instead decided to push forward with the Phase IIIb trial, with an estimated start date of June 30.

In recent years, research into ALS treatments has been ramping up, with companies like Korro Bio, Coya Therapeutics, and QurAlis exploring novel targets against the disease. This development underscores the ongoing efforts to find effective treatments for ALS.

  1. The recent development revolves around BrainStorm Cell Therapeutics, a biotech company, regarding NurOwn's approval for Amyotrophic Lateral Sclerosis (ALS).
  2. A Citizens' Petition was submitted, requesting a reconsideration of NurOwn's approval.
  3. The petition was presented by ALS patients and advocates, featuring new data supporting NurOwn's efficacy.
  4. The new data includes survival data, improvements in biomarkers, and functional outcomes in early-stage ALS patients.
  5. Statistically significant changes in 23 CSF biomarkers, including neurofilament light (NfL), were highlighted, indicating reduced neurodegeneration.
  6. Subgroup analyses showed that NurOwn recipients retained more points on the functional scale compared to placebo groups.
  7. The Food and Drug Administration (FDA) is currently considering the petition, offering an opportunity for a fresh review of NurOwn's data.
  8. BrainStorm Cell Therapeutics supports the petition and welcomes the FDA's willingness to reassess the data.
  9. The company remains committed to advancing access to NurOwn for ALS patients and plans to proceed with a Phase 3b clinical trial.
  10. The ongoing clinical trials include the Phase 3b ENDURANCE trial, designed to enroll 200 early-stage ALS patients.
  11. The trial aims to validate NurOwn's efficacy through a randomised, double-blind, placebo-controlled phase followed by an open-label extension.
  12. The outcome of the FDA's decision will be closely watched, given Commissioner Marty Makary's emphasis on "common sense" approvals for life-threatening diseases.
  13. The decision will depend on how the FDA weighs the new evidence against its stringent approval criteria.
  14. This story has been updated to include further details from BrainStorm on the Phase IIIb trial of NurOwn.
  15. The Phase IIIb trial was initially scheduled to begin last month.
  16. BrainStorm withdrew the Biologics License Application (BLA) prior to its PDUFA date and instead decided to push forward with the Phase IIIb trial.
  17. The estimated start date of the Phase IIIb trial is June 30.
  18. Research into ALS treatments has been ramping up, with companies like Korro Bio, Coya Therapeutics, and QurAlis exploring novel targets against the disease.
  19. This development underscores the ongoing efforts to find effective treatments for ALS.
  20. Science continues to play a critical role in addressing chronic diseases like cancer, chronic respiratory conditions, digestive health issues, and eye health problems.
  21. In the realm of mental health, neurological disorders such as Alzheimer's and Parkinson's are being tackled, along with men's health issues like prostate cancer and erectile dysfunction.
  22. In the face of autoimmune disorders like lupus and multiple sclerosis, research advancements in medical-conditions are paving the way for improved management and treatments.
  23. Climate change has significant implications for health-and-wellness, leading to an increased prevalence of skin conditions, allergies, and respiratory illnesses.
  24. Fitness-and-exercise, skin-care, and nutrition play crucial roles in maintaining overall health and wellness, as well as in weight-management and cardiovascular health.
  25. Aging impacts various aspects of health, from memory and cognitive function to bone density and vision, highlighting the importance of therapies-and-treatments in managing age-related health declines.
  26. Women's health encompasses a wide range of concerns, from reproductive health and sexual health to breast cancer and menopause.
  27. Parenting and child development involve understanding various factors affecting growth and development, from nutrition to mental health.
  28. The intersection of technology, finance, and environmental science presents opportunities for innovative solutions, from investing in clean energy to implementing data-and-cloud-computing for climate modeling.

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