FDA pushes back to December its decision on Sanofi’s tolebrutinib
Developer remains 'confident' of BTK inhibitor's positive impact in MS
The U.S. Food and Drug Administration (FDA) has delayed by three months its decision on whether or not to approve Sanofi’s tolebrutinib as a treatment for multiple sclerosis (MS), pushing back the agency’s expected action date to Dec. 28.
Sanofi is seeking regulatory approval of tolebrutinib for two indications: to treat individuals with nonrelapsing secondary progressive MS (SPMS), and to slow disability accumulation independent of relapse activity in adults with MS. The application had been granted priority review earlier this year, and a decision was originally expected before the end of this month.
The FDA’s extension of the review period follows Sanofi’s submission of additional analyses during this process. The FDA deemed those analyses “a major amendment” requiring extra time for evaluation, the company stated in a press release.
“Sanofi is confident in the potential positive impact tolebrutinib can provide and will continue to collaborate closely with the FDA during the review period,” the company stated.
MS is marked by inflammation that damages healthy cells in the brain and spinal cord. The disease is often marked by relapses, or periods in which symptoms suddenly worsen. Disability may accumulate if patients recover poorly from these attacks. However, most MS patients will also experience disability that gets progressively worse in the absence of relapse activity.
Tolebrutinib won first regulatory approval last month in UAE
Tolebrutinib is a once-daily oral therapy designed to block an enzyme called Bruton’s tyrosine kinase (BTK) that plays a key role in the activation of certain immune cells involved in driving MS.
According to Sanofi, the therapy is specifically designed to target chronic inflammatory processes within the brain and spinal cord that contribute to progressive neurodegeneration and long-term disability in MS.
Sanofi’s application requesting tolebrutinib’s approval in the U.S. was based on data from three completed Phase 3 clinical trials: HERCULES (NCT04411641), GEMINI 1 (NCT04410978), and GEMINI 2 (NCT04410991).
The global HERCULES trial tested tolebrutinib against a placebo in people with nonrelapsing SPMS, with the results showing that the therapy significantly delayed confirmed disability progression by 31% compared with a placebo.
The GEMINI studies, meanwhile, tested tolebrutinib against the approved therapy Aubagio (tolebrutinub) in people with relapsing forms of MS. While those worldwide studies failed to meet their main goal of showing that tolebrutinib could reduce relapse rates compared with Aubagio, data indicated that tolebrutinib significantly reduced the risk of confirmed disability worsening by 29%.
Based on data from the HERCULES study, the therapy was approved to treat nonrelapsing SPMS in the United Arab Emirates last month, marking its first worldwide approval. Sanofi also has applied for tolebrutinib’s approval in the European Union, although the exact indications for which it is requesting an approval in that region have not been disclosed.
Tolebrutinib is also being tested in a fourth Phase 3 trial called PERSEUS (NCT04458051), which is evaluating the treatment candidate in more than 750 people with primary progressive MS. Results are expected in the coming months, according to the developer.
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