March 10, 2009

FDA Allows Formal Testing of Iplex in ALS

The Food and Drug Administration (FDA) announced today that it would work closely with the biopharmaceutical company Insmed of Richmond, Va., to develop a clinical trial of Iplex, an experimental medication developed by the company, in patients with ALS (amyotrophic lateral sclerosis).

This decision represents a change to the agency's previous policy regarding the use of Iplex in ALS, which was that physicians could apply for permission to prescribe the drug for specific patients, a so-called compassionate use program.

The FDA says it will continue to process any requests for compassionate use of Iplex in ALS received by the close of business on March 6 but that subsequent access to the drug for ALS patients will only be through the anticipated clinical trial. For details, see FDA Position on Allowing Patients with ALS Access to Iplex.

About Iplex

Iplex is a combination of insulin-like growth factor 1 (IGF1), a protein that may help protect nerve cells from damage under some circumstances, and IGF1 binding protein 3. Three earlier trials of subcutaneous injections of the IGF1 protein without any binding protein in ALS failed to show a benefit.

Iplex is approved for the treatment of growth failure in children with severe IGF1 deficiency or with a genetic mutation in the gene for growth hormone. However, it is not currently being marketed for this purpose because of a patent dispute.

Insmed is testing Iplex, with MDA support, in a clinical trial to see if it has benefit in type 1 myotonic dystrophy.

The drug has never been formally tested in ALS, although ALS patients in Italy have had access to it, and the Italian regulatory agency has collected data on its use by 110 people. The FDA has recently obtained these data, from which no conclusions could be drawn about efficacy but which suggest the compound is reasonably safe in this disease.

For details abut the upcoming Insmed clinical trial, contact Insmed at (804) 565-3083 or iplex_als@insmed.com.

About ALS

ALS is a disease in which muscle-controlling nerve cells in the brain and spinal cord are lost, resulting in rapidly progressive paralysis and usually in death within three to five years of symptom onset. The disease is inherited approximately 10 percent of the time. In the other 90 percent of cases, the cause remains unknown. MDA is the world’s largest nongovernmental funder of ALS services and research.