Tolebrutinib gets UAE OK for progressive MS, a world 1st
FDA decision on MS therapy expected in September
The United Arab Emirates became the first country to approve tolebrutinib, clearing it to treat adults with nonrelapsing secondary progressive multiple sclerosis (SPMS).
Sanofi’s oral BTK inhibitor is also the first approved therapy that directly addresses the chronic inflammation that contributes to disability progression independent of relapses.
“The approval of tolebrutinib marks a transformative step in addressing one of the most complex neurological diseases,” Fatima Al Kaabi, director-general of the Emirates Drug Establishment, said in a press release from the Emirates News Agency. “It embodies our commitment to accelerating access to innovative therapies that make a real difference in patients’ lives.”
The U.S. Food and Drug Administration (FDA) is reviewing tolebrutinib for the same indication, and as a potential treatment to slow disability progression independent of relapse activity in adults with MS. A regulatory decision is expected by Sept. 28.
Tolebrutinib works by blocking the activity of Bruton tyrosine kinase (BTK). This enzyme is needed to activate several immune cells, including B-cells and microglia, that play a central role in MS.
Slowing disability progression
While some approved MS therapies can delay disability progression that occurs as a result of poor recovery from relapses, none have been shown to slow the progression that takes place independent of relapses.
However, recent data from three Phase 3 clinical trials have suggested that tolebrutinib may do just that. Results from the HERCULES trial (NCT04411641), which enrolled 1,131 adults with nonrelapsing SPMS, showed that about 2.5 years of tolebrutinib treatment led to a 31% lower risk of disability progression compared with a placebo.
Over the course of the trial, 22.6% of patients given tolebrutinib and 30.7% of those on the placebo experienced six-month confirmed disability progression, defined as an increase in EDSS scores that is sustained for at least six months.
Similar results were seen in the GEMINI 1 (NCT04410978) and GEMINI 2 (NCT04410991) trials, which enrolled 1,873 adults with relapsing forms of MS and randomly assigned them to receive tolebrutinib or Aubagio (teriflunomide).
While tolebrutinib failed to outperform Aubagio at reducing relapse rates, the proportion of patients who experienced six-month confirmed disability worsening was significantly lower with tolebrutinib (8.3% vs. 11.3%), reflecting a 29% reduced risk of disability worsening.
Al Kaabi said the approval “is the result of collaborative efforts between the Establishment and its international partners to support scientific innovation and translate it into real-world patient benefit.”
“At the Emirates Drug Establishment, we believe in fast-tracking regulatory processes in areas of high medical need,” she said. “This achievement reaffirms the readiness of our healthcare system to keep pace with rapid scientific advancements and deliver high-quality treatments that enhance quality of life and strengthen national health security.”
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