When is Edavarone (generic name: Edasalonexent not applicable, as Edavarone is the generic) indicated for patients with Amyotrophic Lateral Sclerosis (ALS)?

Edaravone in Amyotrophic Lateral Sclerosis (ALS)

Edaravone is indicated for patients with ALS who are in the early stages of disease with preserved respiratory function, particularly those with a disease duration of less than 2 years and with mild to moderate functional decline. Based on the available evidence, edaravone has been shown to slow the progression of ALS in specific patient populations.

Patient Selection Criteria for Edaravone

• Edaravone is most beneficial for patients who meet the following criteria:

  • Disease duration of ≤3 years 1
  • Forced vital capacity (FVC) ≥70% of predicted value 1
  • Mild to moderate functional impairment 2

• Patients in early stages of ALS show better response to edaravone therapy, with studies demonstrating a 33% reduction in the rate of physical function decline compared to placebo 3

Administration and Formulations

• Edaravone is available in two formulations:

  • Intravenous (IV) formulation: administered as a 60 mg infusion over 60 minutes in 28-day cycles 3
  • Oral suspension (105 mg): follows the same dosing schedule as IV formulation but with improved convenience for patients 4, 1

• Dosing schedule follows a specific pattern:

  • First cycle: daily administration for the first 14 days
  • Subsequent cycles: 10 days of administration during the first 14 days of each 28-day cycle 2

Efficacy and Outcomes

• Edaravone works as a potent free radical scavenger and antioxidant, preventing oxidative stress-induced motor neuron death in ALS patients 3

• Clinical studies have demonstrated that edaravone can slow the decline in ALSFRS-R scores (ALS Functional Rating Scale-Revised), though the effect size varies between studies 2

• Long-term treatment (up to 144 weeks) has been studied with the oral formulation, showing sustained safety profile 5

Safety Profile

• Most common adverse events associated with edaravone treatment include:

  • Fall (22.2%)
  • Muscular weakness (21.1%)
  • Constipation (17.8%)
  • Fatigue, dizziness, and headache 1

• Serious adverse events are generally related to disease progression rather than the medication itself 1, 5

• Both IV and oral formulations demonstrate similar safety profiles, with no new safety concerns identified in long-term extension studies 5

Monitoring and Follow-up

• Regular assessment of functional status using ALSFRS-R is recommended to evaluate treatment response 2

• Respiratory function monitoring (FVC) should be performed periodically to assess disease progression 1

• Treatment may be continued as long as the patient shows benefit or until disease progression makes treatment futile

Practical Considerations

• The oral suspension formulation provides a more convenient alternative to IV administration, especially for patients with limited mobility or those who live far from infusion centers 4

• Edaravone can be administered via percutaneous endoscopic gastrostomy (PEG) tube for patients with dysphagia, with similar pharmacokinetic profiles to oral administration 4

• Treatment should be initiated as early as possible after diagnosis to maximize potential benefits in slowing disease progression 3, 1