The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to tolebrutinib for the treatment of non-relapsed secondary progressive multiple sclerosis (SPMS) in adults. This designation is based on positive results from the phase III HERCULES study , which demonstrated that tolebrutinib prolonged the time to confirmed disability progression by 31% compared to placebo (HR 0.69; 95% CI: 0.55-0.88; p=0.0026). Further analysis of secondary endpoint data further showed that nearly twice as many participants (10%) treated with tolebrutinib experienced a slowdown in confirmed disability progression compared with those treated with placebo (5%) (HR 1.88; 95% CI: 1.10 to 3.21; nominal p-value = 0.021).

In the United States, Breakthrough Medicine designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. Drugs that meet the criteria for this designation must have demonstrated preliminary clinical evidence that they provide significant improvements in a number of important clinical outcomes compared to other available drugs.

“The Breakthrough Medicine designation demonstrates that tolebrutinib has the potential to delay disability progression and thus address a critical medical need for people with multiple sclerosis. We look forward to working with the FDA in the regulatory review of this first-in-class medicine for the treatment of relapse-free secondary progressive multiple sclerosis, as there are currently no approved medicines for this indication.”

Elevations of liver transaminases greater than 3 times the upper limit of normal (>3xULN) were observed in 4.1% of participants treated with tolebrutinib compared with 1.6% in the placebo group. In the tolebrutinib group, a small proportion of participants (0.5%) experienced elevations of ALT greater than 20 times the upper limit of normal (>20xULN), all within the first 90 days of treatment. All but one case of elevated liver transaminases resolved without medical intervention. Implementation of more frequent monitoring helped mitigate the risk of serious hepatic sequelae. 

Regulatory submissions are being finalized in the United States and are ongoing in the European Union. As with all medicines, Sanofi expects to confirm acceptance of regulatory submissions for tolebrutinib. The Phase 3 PERSEUS study in primary progressive MS is ongoing and results are expected in the second half of 2025.

Tolebrutinib is currently in clinical development and its safety and efficacy profiles have not been evaluated by any regulatory authority.

 

Source: globenewswire.com

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