ICER to Assess Sanofi’s Tolebrutinib for Secondary Progressive MS

The Institute for Clinical and Economic Review (ICER) has announced plans to assess the clinical efficacy and value of tolebrutinib in the treatment of secondary progressive multiple sclerosis (SPMS). Public discussion and review panel voting is planned for June 2025.

Tolebrutinib is an investigational Bruton’s tyrosine kinase (BTK) inhibitor being developed by Paris-based Sanofi for the treatment of relapsing forms of MS as well as nonrelapsing SPMS (nrSPMS). Currently, there is no approved treatment for this form of MS. The oral brain penetrant performed well in the phase 3 HERCULES trial, which met the primary end point of delaying time to onset of confirmed disability progression in patients with nrSPMS. Sanofi plans to file for FDA approval by the end of the year.

ICER will assess the health and economic outcomes of tolebrutinib for nrSPMS compared to supportive care, including pharmacological and nonpharmacological treatments targeting MS symptoms not treated with disease-modifying treatments. These can include pain, numbness, fatigue, urinary incontinence and issues with cognitive function.

Objectives will include patient-related outcomes, such as disability progression, quality of life, cognitive function, depression and adverse events. Another outcome of interest is liver toxicity, with has plagued clinical trials of several BTK inhibitors, including tolebrutinib. MRI activity and disease impact on caregivers will also be assessed.

Evidence for treatment effectiveness will be derived from randomized trials, systematic reviews, and cohort studies. ICER will also consider input from patients and patient advocates, information gathered from regulatory documents, and other input from manufacturers.

The report will also include a lifetime cost-effectiveness analysis of tolebrutinib versus best supportive care in adults with nrSPMS. The analysis will include direct medical costs, such as drug administration and monitoring, and indirect costs, such as factors affecting patient and caregiver productivity.

Lastly, the review will include an assessment of benefits beyond health and special ethical priorities. These factors include whether there is a substantial unmet treatment need, the condition is of significant relevance to racial or ethnic groups that have been affected by disparities in healthcare systems, the treatment is likely to improve the caregiver’s quality of life, or the treatment’s mechanism of action or delivery method has the potential to improve treatment access.