Sanofi (NASDAQ: SNY) has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its Bruton’s tyrosine kinase (BTK) inhibitor, Tolebrutinib, for the treatment of adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS).
Phase III HERCULES Study Results Influence FDA Decision
The FDA’s decision is supported by the positive outcomes from the Phase III HERCULES study. The study demonstrated that tolebrutinib significantly delayed the time to onset of 6-month confirmed disability progression (CDP) by 31% when compared to a placebo. The hazard ratio was 0.69 with a 95% confidence interval of 0.55-0.88, and the result was statistically significant with a p-value of 0.0026.
Tolebrutinib’s Efficacy in Disability Improvement
Further analysis of the HERCULES study revealed that the number of participants who experienced confirmed disability improvement was nearly double in the tolebrutinib group (10%) compared to those on a placebo (5%). This highlights the potential of tolebrutinib to not only slow the progression of nrSPMS but also to contribute to the improvement of disability in some patients.
Tolebrutinib’s Unique BTK Inhibitor Profile
Tolebrutinib is recognized as the first and only brain-penetrant BTK inhibitor for MS with Breakthrough Therapy Designation by the FDA. This status underscores the drug’s potential to offer a novel treatment option for patients with nrSPMS and addresses an unmet need in the treatment landscape of this debilitating disease.-Fineline Info & Tech
