Long-term Tecfidera slows MS disability progression in large trial
New analysis shows approved therapy also significantly reduces relapse rates
Treatment with Tecfidera (dimethyl fumarate) significantly reduces relapse rates for people with multiple sclerosis (MS), and most MS patients on the approved therapy remain free from disability progression for several years.
That’s according to a final analysis from the Phase 4 ESTEEM clinical trial (NCT02047097), which tracked outcomes from Tecfidera treatment among more than 5,100 MS patients. The results also did not reveal any unexpected safety issues with the long-approved therapy, the researchers noted.
“The ESTEEM study, in which over 5,000 patients received [Tecfidera] in a real-world setting for up to 6.5 years, indicated sustained effectiveness of [Tecfidera] and safety consistent with the well-known profile of [Tecfidera],” the team wrote.
The study, titled “Long-Term Safety and Effectiveness of Dimethyl Fumarate in Patients with Multiple Sclerosis Treated in Routine Medical Practice: Final Analysis of the ESTEEM Study,” was published in Neurology and Therapy. The work was funded by Biogen, which markets name-brand Tecfidera. Several generic versions of the therapy also are available.
Researchers call ESTEEM ‘largest and longest real-world setting study’
Tecfidera has been approved in the U.S. as a treatment for relapsing types of MS for more than a decade. The therapy’s mechanism of action isn’t fully understood, but it’s broadly thought to work by modulating the activity of immune cells, thereby limiting the inflammation in the brain and spinal cord that drives MS.
The ESTEEM study, completed in 2022, tracked outcomes from 5,124 patients who were treated at nearly 400 sites around the globe with at least one dose of the medication. The study’s main goal was to evaluate safety outcomes, but the treatment’s effect on MS relapses and disability progression also were assessed.
Almost all (98.9%) of the study’s participants were specifically diagnosed with relapsing-remitting MS (RRMS). The study included nearly 1,500 people who started on Tecfidera after being newly diagnosed with MS, and about 2,200 who had previously been on interferon treatments or glatiramer acetate (sold as Copaxone and others).
The average duration of Tecfidera treatment was 31 months, or about 2.5 years, though some patients had more than six years of follow-up data available.
“The clinical effectiveness results reported here are, to our knowledge, from the largest and longest real-world setting study of [Tecfidera],” the researchers noted.
MS relapse rates dropped by 90% for patients on Tecfidera
About half (51%) of the participants stopped taking the medication at some point during the study, with nearly 1 in 5 (22%) of them stopping Tecfidera specifically due to safety issues. The most common safety issues leading to treatment discontinuation were digestive complaints and low counts of immune cells, both of which are well-established side effects of Tecfidera.
Serious safety issues were documented in 8%, or fewer than 400, of the patients on Tecfidera. The most common serious safety problems were infections. The researchers highlighted, however, that there was not a clear connection between low counts of immune cells and infections while on treatment.
Compared with rates from before starting on Tecfidera, relapse rates while on the medication were reduced by 90.1%, from 0.81 relapses per year at the study’s start to 0.08 relapses per year over six years of treatment.
Data after four years also indicated that 87.6% of patients had not experienced disability worsening that was sustained for at least 48 weeks, or nearly one year. After six years, the rate of patients without sustained disability progression was similar, at 87%.
In fact, about 16.7% of participants had experienced sustained improvements in disability, meaning their symptoms were less severe, after six years.
Safety and efficacy outcomes were broadly similar across all subgroups of patients, including those who were newly diagnosed, those who had previously been on other MS treatments, and patients with milder disease. Likewise, analyses from the study overall also have indicated that the safety and efficacy of Tecfidera are consistent across racial and ethnic groups.
The data reported in ESTEEM were overall comparable with findings from other, shorter studies of Tecfidera in MS patients.
“These data demonstrate that [Tecfidera] is an effective treatment option in the real-world setting, with a well-known safety record,” the scientists concluded.
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