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Real-world data fail to show a benefit of edaravone in ALS

JAMA Neurology
Reuters Health - 10/01/2022 - Treatment with edaravone did not provide a clinically relevant benefit on progression of amyotrophic lateral sclerosis (ALS) over standard therapy alone in a real-world cohort study conducted in Germany.

Intravenous edaravone is approved as a disease-modifying drug for ALS, but evidence for efficacy is limited to short-term beneficial effects shown in one study from Japan in a subpopulation in which efficacy was expected, researchers explain in JAMA Neurology.

Dr. Simon Witzel of Ulm University and colleagues evaluated the safety and long-term effectiveness of edaravone in an observational study of ALS patients from 12 academic centers affiliated with the German Motor Neuron Disease Network (MND-NET).

Disease progression among 116 patients treated for a median of 13.9 months with edaravone did not differ from 116 patients treated for a median of 11.2 months with standard therapy, which included riluzole in the vast majority, the researchers report.

Even in a subgroup of matched patients that would have been expected to benefit based on the Japanese trial, edaravone did not have disease-modifying benefit over standard therapy, they say.

Edaravone also did not significantly prolong time to ventilation or survival probability.

"Although we primarily evaluated outcomes associated with long-term treatment, additional analyses in our study provided no evidence that edaravone treatment was associated with a temporary, short-term benefit," the researchers say.

Dr. Jonathan Glass and Dr. Christina Fournier with the department of neurology at Emory University in Atlanta offer perspective on the German observational study in a linked editorial.

They note that the approval of edaravone by the U.S. Food and Drug Administration in 2017 was "somewhat of a surprise to many in the ALS scientific and clinical research communities but was seen by patients as new hope for slowing disease."

"Although the results of an observational trial may be criticized as being less trustworthy than a prospective placebo-controlled trial, the rigor of this current virtual trial makes it both impressive and believable," they write.

"The total number of edaravone-treated patients is three times the number included in the Japanese trial, and the number of subgroup patients and controls was equal to that in the original positive study. These new data certainly add to the conversation about the usefulness of edaravone for the treatment of patients with ALS," Dr. Glass and Dr. Fournier say.

Looking at the bigger picture, they note that "all ALS and neurodegenerative disease stakeholders are hopeful that therapies that meaningfully improve the lives of patients will soon be discovered, and when this happens, any regulatory or bureaucratic barriers should be removed to bring the drug to market as soon as possible."

"Incremental progress is important in drug development and should be celebrated; however, accelerated approval of marginally helpful drugs does not bring us closer to the long-term goal of developing meaningful treatments to help patients with devastating diseases," the editorialists conclude.

The study had no specific funding.

SOURCE: https://bit.ly/3f6qnRZ and https://bit.ly/3r5Hp8o JAMA Neurology, online January 10, 2022.

By Reuters Staff

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