Drugs

Sanofi Scraps Tolebrutinib MS Filing after Phase 3 Miss

Sanofi says it will not seek FDA approval for tolebrutinib in primary progressive multiple sclerosis (PPMS) after the drug failed to meet its primary endpoint in a pivotal Phase 3 trial, dealing a setback to the company’s ambitions for the brain-penetrant BTK inhibitor even as regulatory review continues in another progressive MS population.

Results from the PERSEUS Phase 3 study showed that tolebrutinib did not significantly delay time to six-month composite confirmed disability progression compared with placebo in PPMS patients, which accounts for roughly 10% of the MS population. Based on those data, Sanofi said it will not pursue registration for PPMS.

“We are disappointed by today’s results,” Sanofi R&D head Houman Ashrafian is quoted in a release as saying, adding that the findings will help advance understanding of MS biology. He emphasized that the company remains confident in the potential role of tolebrutinib in non-relapsing secondary progressive multiple sclerosis (nrSPMS), where disability continues to worsen in the absence of relapses.

In a separate update, Sanofi says FDA’s ongoing review of tolebrutinib for nrSPMS is now expected to extend beyond the user fee review action target date of 12/28. The company now anticipates further guidance from the agency by the end of the first quarter of 2026.

Sanofi says it has submitted an expanded access protocol at the agency’s request, allowing eligible nrSPMS patients potential access to the investigational therapy while the review continues.

The company says tolebrutinib is designed to penetrate the brain and inhibit Bruton’s tyrosine kinase, targeting chronic, “smoldering” neuroinflammation thought to drive disability progression in MS. Addressing disability accumulation remains one of the largest unmet needs in the disease, particularly in progressive forms where treatment options are limited, according to the company.

Despite the PPMS disappointing news, Sanofi says it remains committed to advancing tolebrutinib and its broader neurology pipeline, which includes multiple Phase 3 programs across neuro-inflammatory and neuro-degenerative diseases.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

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FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome