FDA Grants Breakthrough Therapy Designation to Sublingual Treatment for Acute Ischemic Stroke

Sanbexin (edaravone and dexborneol; Simcere, Nanjing, China) sublingual tablets have been granted Breakthrough Therapy designation by the Food and Drug Administration (FDA) for the treatment of acute ischemic stroke (AIS). The designation was granted based on positive data from the TASTE-SL phase 3 clinical trial (NCT04950920), in which people with AIS treated with Sanbexin showed statistically significant improvements in functional outcomes compared with those who received placebo. The results of TASTE-SL were published in JAMA Neurology.

Sanbexin is a formulation of edaravone and dexborneol, which according to a statement from Simcere, have synergistic antioxidant and anti-inflammatory effects which reduce impairment after AIS. The sublingual route of administration enables flexible treatment outside of the hospital, potentially reducing disability and improving outcomes.

In TASTE-SL, a clinical study conducted in China, 914 study participants with AIS were randomly assigned to receive either Sanbexin (n=450) at 36 mg (30 mg of edaravone, 6 mg of dexborneol) or placebo (n=464) twice daily for 14 days, with 90 days of follow up. The primary efficacy outcome was the proportion of participants with a modified Rankin Scale (mRS) score of ≤1 on day 90 after randomization.

• The primary efficacy outcome was reached in 64.4% of participants in the Sanbexin group vs 54.7% of participants in the placebo group (risk difference, 9.70%; 95% CI, 3.37% to 16.03%; odds ratio, 1.20; 95% CI, 1.15 to 1.95; P=.003)
• Sanbexin had a consistent effect across multiple subgroups according to the results of subgroup analysis.
• There was a 89.8% risk of adverse events in those assigned to the Sanbexin group vs a 90.1% in the placebo group.

Breakthrough Therapy designation is intended to expedite the development and review of drugs that treat serious conditions with positive preliminary clinical evidence. Products that receive Breakthrough Therapy designation are granted all the features of Fast Track designation as well as guidance in an efficient drug development program.