FDA recognizes the critical unmet medical need for new, effective treatments for amyotrophic lateral sclerosis (ALS). We are committed to working with drug companies and the ALS community to facilitate development and approval of drugs to treat this devastating disease. FDA is prepared to use all expedited development and approval pathways available to us to further this mutual goal.
FDA knows that ALS patients, their families, and others in the ALS community are concerned about the status of Genervon’s experimental drug, GM604, for the treatment of ALS. However, FDA is prohibited by law, under usual circumstances, from releasing confidential information about experimental drugs, including GM604.
We call upon Genervon to release all the data from their recently completed trial in order to allow a more informed discussion of the trial findings among ALS stakeholders. Such a release should include the pre-specified clinical outcome measures as assessed by change from baseline observations that were taken just prior to randomization to drug or placebo. Such data provide the strongest basis to assess for drug-related changes in efficacy and safety parameters.
FDA will continue to provide detailed advice and support to Genervon as they pursue further study of GM604 to determine if it is safe and effective to treat ALS. We remain committed to working with the ALS community to find effective treatments for this disease.