FDA Grants “Compassionate Use” Access to Drug for ALS Patient: An Overview of the Issues Surrounding Expanded Access to Experimental Drugs

On March 10, 2009, the Food and Drug Administration reversed course and announced it will now grant patients suffering from ALS (Amyotrophic Lateral Sclerosis, or “Lou Gehrig’s Disease) access to Iplex on a compassionate use basis. ALS is a fatal neurodegenerative disease for which there is no known cure. Iplex is currently approved by the FDA only for the treatment of growth failure in children with severe primary IGF-1 deficiency or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH. While Iplex’s efficacy for ALS has not been established by any well-controlled clinical studies, there is substantial anecdotal evidence that the drug has been helpful when administered to ALS patients in Italy.

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