How does IV edaravone fare for ALS in the real world?

Intravenous (IV) edaravone seems to continue to provide hope for individuals with amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease with no known cure, according to data presented at AAN 2022.

“[We sought] to evaluate overall survival (OS) in patients with ALS treated with IV edaravone compared with those who were not treated with IV edaravone,” said Dr Benjamin Brooks from Clinical Trials Planning LLC, Charlotte, North Carolina, US, and colleagues. “[In our] real-world analysis … continued IV edaravone treatment in a large predominantly riluzole-treated US cohort is associated with improved OS compared with not using IV edaravone.”

The team evaluated ALS patients who initiated IV edaravone treatment between August 2017 and March 2020 and were enrolled in a US administrative claims database. Propensity score matching identified IV edaravone (cases; n=318) and non-edaravone recipients (controls; n=318) matched for covariates* that could influence survival. About two-thirds (65 percent) of participants in both arms were once prescribed riluzole. [AAN 2022, abstract P2.004]

As of March 2021, there were more deaths among those who have not been treated with IV edaravone as opposed to those who have been receiving the drug (61.6 percent vs 48.7 percent).

Median OS was longer among cases vs controls (29.5 and 23.5 months, respectively), with the risk of death being 27-percent lower among edaravone vs non-edaravone recipients (hazard ratio, 0.73, 95 percent confidence interval, 0.59–0.91; p=0.005).

In another real-world analysis, de-identified data from providers of home/alternative-site infusions and specialty pharmacy services across the US that were treating ALS patients receiving IV edaravone were analysed. The analysis included 455 participants (52.1 percent male, 33.6 percent between 60 and 69 years at the start of treatment at the centre) who have received their first IV edaravone dose between August 2017 and December 2020. [AAN 2022, abstract P2.005]

At the beginning of treatment, mean ALS Functional Rating Scale-Revised and Forced Vital Capacity scores were 34.3 percent and 70.7 percent, respectively.

By the end of the study period, nearly half (47.6 percent) discontinued treatment, primarily owing to death or hospice (35.0 percent). This suggests that half of the cohort are still on treatment. The other reasons for treatment withdrawal were patient’s choice (27.6 percent), change in care or insurance (19.4 percent), and doctor’s choice (9.2 percent). Other unspecified reasons accounted for 8.8 percent.

“[We wanted to] describe treatment outcomes in ALS patients treated with IV edaravone since FDA approval … [as] information on its real-world effectiveness is limited,” said Dr Melissa Hagan from Mitsubishi Tanabe Pharma America, Inc, Jersey City, New Jersey, US. “[We hope] that this information will be useful to clinicians who prescribe IV edaravone for their patients with ALS.”

Based on evidence demonstrating its ability to slow the rate of physical functional decline, IV edaravone has gained US FDA approval in May 2017. It is one of only two drugs approved for ALS treatment in the US.

An oral formulation of edaravone is currently being investigated.