Minimal Residual Disease Endpoint Would Aid Leukemia Drug Development, Experts Say
An international panel of hematology experts tells an FDA/ASCO workshop Feb. 27 that minimal residual disease would be a useful surrogate endpoint for assessing the effects of chronic lymphocytic leukemia therapies because it has been strongly correlated with longer-term outcomes in the disease.
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More work needs to be done to validate MRD as predictive of clinical outcomes in hematological malignancies before it can be used as the basis for accelerated approval, FDA and other stakeholders say.
A Pink Sheet timeline shows the regulatory highs and lows of AMX0035's path to US approval for treatment of amyotrophic lateral sclerosis.
Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’
The single positive Phase II CENTAUR study, along with confirmatory evidence of benefit on long-term survival in the open-label extension, demonstrate substantial evidence of effectiveness, the agency says, acknowledging a level of uncertainty that is acceptable in the disease context.