Sanofi, the prominent French pharmaceutical company, disclosed on Friday that the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Therapy designation to tolebrutinib for the treatment of adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS).
Tolebrutinib stands as the only brain-penetrant BTK inhibitor for multiple sclerosis (MS) to receive such a designation from the FDA. This decision was grounded in favorable outcomes from the HERCULES phase 3 study. The study evidenced that tolebrutinib postponed the commencement of the 6-month confirmed disability progression (CDP) by 31% compared to a placebo. Additionally, secondary endpoint analyses revealed nearly double the number of participants experienced confirmed disability improvement with tolebrutinib than with placebo.
The FDA's Breakthrough Therapy designation aims to accelerate the development and assessment of drugs in the U.S. designed to treat severe or life-threatening conditions.
Erik Wallstrm, Sanofi’s Global Head of Neurology Development, remarked, "This Breakthrough Therapy designation underscores tolebrutinib's potential to delay disability progression, addressing a critical unmet need for multiple sclerosis patients. We are eager to collaborate with the FDA during the review of this pioneering medication for non-relapsing secondary progressive multiple sclerosis, an area currently lacking approved treatments."
Sanofi is in the final stages of regulatory submissions for tolebrutinib in the U.S. and is also preparing for submission in the European Union. As is customary, Sanofi will confirm once their regulatory submission has been accepted.
Meanwhile, the PERSEUS phase 3 study focusing on primary progressive MS is in progress, with results expected in the second half of 2025.
It is important to note that tolebrutinib is still undergoing clinical investigation, and its safety and efficacy are yet to be evaluated by any regulatory body.
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