Investigational, Once-Daily Pill for ALS Receives FDA Fast Track Designation

The Food and Drug Administration (FDA) has granted Fast Track designation to tazbentetol (SPG302; Spinogenix, Los Angeles, CA), an investigational once-daily oral therapy under development for the treatment of amyotrophic lateral sclerosis (ALS). Fast Track designation is intended to support more frequent FDA interaction and potential expedited development and review for therapies addressing serious conditions with unmet medical needs.

Tazbentetol previously received FDA Orphan Drug designation for ALS in 2021. According to a statement from Spinogenix, the therapy is designed to promote synaptic restoration by targeting glutamatergic synapses, an approach the company is also evaluating in other neurologic and neuropsychiatric conditions, including Alzheimer disease and schizophrenia.

The FDA’s decision was based on data from a phase 2a study of tazbentetol in ALS (NCT05882695). In the study, tazbentetol demonstrated a favorable safety and tolerability profile and preliminary functional findings based on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).

Key Findings

• At the end of treatment, 82% of participants exhibited a stable or improved rate of decline on ALSFRS-R.
• On average, of those 82% of participants, 76% showed a slower rate of decline through 6 months compared with matched historical controls from the PRO-ACT database.
• Quantitative electroencephalogram (EEG) findings showed changes in ALS-associated patterns that the company described as supportive of the functional findings.

Source

Spinogenix. Spinogenix receives U.S. FDA Fast Track Designation for tazbentetol for the treatment of ALS. PR Newswire. Published May 5, 2026. Accessed May 6, 2026. https://www.prnewswire.com/news-releases/spinogenix-receives-us-fda-fast-track-designation-for-tazbentetol-for-the-treatment-of-als-302762461.html