(A-303) Evolution of the treatment landscape for patients diagnosed with amyotrophic lateral sclerosis in the United States

Background: Treatment options for ALS aim at modifying disease, alleviating symptoms, and improving quality of life. Over the past decade, United States Food and Drug Administration (FDA)-approved treatments for ALS have evolved.

Objectives: To describe the evolution of the treatment landscape for patients diagnosed with amyotrophic lateral sclerosis (ALS).

Methods: Patients aged ≥18 years with incident ALS between January 2016 through December 2022 were identified using the Inovalon closed claims database sourced through HealthVerity. Patients were required to have continuous enrollment for 6 months before and after incident ALS diagnosis. FDA-approved ALS treatments, including riluzole, Mitsubishi Tanabe Pharma America (MTPA) IV (intravenous) and oral edaravone (Radicava® IV and Radicava ORS®), and sodium phenylbutyrate and taurursodiol (withdrawn from the US market following this analysis period), initiated within 6 months of ALS diagnosis and on/after the incident ALS diagnosis were examined by ALS diagnosis year.

Results: We observed an increase in the use of riluzole among patients with ALS diagnosed between 2016 and 2022, from 5.9% in 2016 to 24.8% in 2022. MTPA IV edaravone was introduced in 2017, and its use increased from 1% to 4.8% between 2017 and 2021. However, with the introduction of MTPA oral edaravone in 2022, use of MTPA IV edaravone decreased to 1%, while use of MTPA oral edaravone increased to 8.9%. Over the study period, a decrease was observed in untreated patients with ALS, from 87.1% in 2016 to 70.7% in 2022.

Conclusions: During the 2016-2022 period, there was increased use of riluzole and a slow shift toward MTPA IV edaravone. With FDA approval of MTPA oral edaravone in 2022, there was a shift observed toward this newer option. Although many patients still had not received any FDA-approved treatments, that number has decreased, possibly due, in part, to the approval of MTPA oral edaravone.