ICER Midwest CEPAC Public Meeting on AMX0035 and Oral Edaravone for Amyotrophic Lateral Sclerosis

• AMX0035 (Amylyx Pharmaceuticals, Inc.)
• oral edaravone (Mitsubishi Tanabe Pharma Development America, Inc.)

Given current data, AMX0035 is comparable or better to standard of care; AMX0035 would achieve common thresholds for cost-effectiveness if priced between $9,100 – $30,600 per year. Evidence indicates oral edaravone is comparable or incrementally better to standard of care; treatment would achieve common thresholds for cost-effectiveness if priced between $1,400-$3,200 per year

ICER’s Chief Medical Officer, David Rind, MD stated:

“There is tremendous need for new therapies in ALS. Current treatment of this devastating disease is largely focused on supportive care. AMX0035 appears to extend life and modestly slow disease progression, but we expect vigorous debate at the public meeting on the strength of evidence, which hinges largely on the findings from a single trial, particularly given the negative vote by the FDA Advisory Committee.

We also anticipate a policy discussion on whether, should this drug receive FDA approval, the degree of uncertainty should be reflected in a price scaled closer to the cost of production pending completion of trials that can confirm the magnitude of the benefit for patients.  In this report we also evaluated the evidence on an oral formulation of edaravone.  The oral version is much less burdensome to patients than the intravenous formulation, which is likely to lead to increased use, but the price of edaravone appears too high to align reasonably with the benefits it provides.”