An FDA advisory panel voted against the effectiveness of BrainStorm Cell Therapeutics’ NurOwn in treating mild-to-moderate Amyotrophic Lateral Sclerosis (ALS). The panel voted 17 to 1 against the therapy being shown to be effective, with one panelist abstaining from the voting. While the FDA is not obligated to follow the recommendations of its expert panels, it generally does so. The agency is expected to make a decision regarding the approval of the cell therapy by December 8. Shares of BrainStorm were halted in anticipation of the vote and remained halted in after-hours trading.
BrainStorm Cell Therapeutics’ stock experienced a significant drop earlier this week after FDA reviewers expressed concerns about the drug’s effectiveness in briefing documents for the advisory panel meeting. The Phase 3 trial for NurOwn failed to meet its primary or secondary endpoints, but the company attributed this outcome to a “floor effect” that can complicate the measurement of disease progression in more advanced patients. However, subsequent data analysis suggested that the therapy showed clinically meaningful effectiveness in patients with less advanced disease.
Although the FDA advisory panel’s vote against the effectiveness of NurOwn is a setback for BrainStorm, the final decision still lies with the FDA itself. The agency will consider the panel’s recommendation along with other factors before reaching a decision by December 8. The halt in trading for BrainStorm’s stock indicates the significance of this decision for the company. Investors are eagerly awaiting the FDA’s decision and its potential impact on the future of NurOwn and its potential in treating ALS.