What are the indications for Edavarone (Radicut) in Amyotrophic Lateral Sclerosis (ALS)?

Indications of Edaravone in ALS

Edaravone is indicated for patients with amyotrophic lateral sclerosis (ALS) who meet specific criteria, particularly those with early-stage disease, preserved respiratory function, and definite or probable ALS diagnosis within two years of symptom onset to slow the rate of physical functional decline.

Patient Selection Criteria for Edaravone Treatment

• Edaravone demonstrated efficacy in a subgroup of ALS patients with the following characteristics:

  • Definite or probable ALS according to El Escorial revised Airlie House diagnostic criteria 1
  • Disease duration ≤2 years 1, 2
  • Forced vital capacity (FVC) ≥80% of predicted 1, 2
  • ALSFRS-R score ≥2 points for all item scores before treatment (indicating relatively preserved function) 1, 2

• Clinical trials showed that patients meeting these criteria experienced significantly slower decline in ALSFRS-R scores compared to placebo (p=0.0270) 1

Administration and Formulations

• Edaravone is available in two formulations:

  • Intravenous formulation (original approved form) 3
  • Oral suspension formulation (more recently FDA-approved) 3, 4

• The approved dosing regimen follows an on/off cycle pattern:

  • Initial treatment cycle: daily dosing for 14 days followed by a 14-day drug-free period
  • Subsequent treatment cycles: daily dosing for 10 days out of 14-day periods, followed by 14-day drug-free periods 5
  • Research has shown that daily dosing does not provide superior efficacy compared to the approved on/off regimen 5

Special Considerations for Bulbar Dysfunction

• For patients with bulbar dysfunction:
  • Regular monitoring for dysphagia progression is essential as it may affect medication administration 6
  • Alternative administration via percutaneous endoscopic gastrostomy (PEG) tube is possible when oral administration becomes difficult 4
  • Nutritional status should be closely monitored, as weight loss negatively impacts survival 6

Efficacy and Safety Profile

• Edaravone slows the rate of physical functional decline in ALS patients who meet the specific criteria 5, 2

• Long-term safety data from a 48-week extension study showed:

  • No sudden deterioration in ALSFRS-R scores with continued treatment 2
  • Common adverse events include constipation, dysphagia, and contusion 2
  • For oral formulation: fall (22.2%), muscular weakness (21.1%), and constipation (17.8%) were the most common treatment-emergent adverse events 3

• The oral suspension formulation demonstrated a favorable safety profile with no new safety concerns identified after 48 weeks of treatment 3

Clinical Pearls and Pitfalls

• Edaravone should be initiated early in the disease course (within 2 years of symptom onset) for optimal benefit 1, 2

• Regular monitoring of respiratory function is essential as treatment is most beneficial in patients with preserved respiratory function (FVC ≥80%) 1, 2

• Patients should be informed that edaravone slows but does not stop disease progression 2

• The medication should be administered according to the approved on/off dosing regimen, as continuous daily dosing has not shown superior efficacy 5