Italians With ALS Gain Access to Drug

The Italian Ministry of Health recently asked Insmed, a Richmond, Va., biopharmaceutical company, to make its drug Iplex available to amyotrophic lateral sclerosis (ALS) patients in Italy. The request has been granted.

Iplex is a combination of insulin-like growth factor 1 (IGF1) and IGF binding protein 3. It’s approved by the U.S. Food and Drug Administration to treat certain types of growth failure in children, and it’s also being tested in people with myotonic muscular dystrophy

Almost all IGF1 that circulates in the bloodstream is attached to IGF binding protein 3, so the combination may be a better mimic of human physiology than IGF1 alone.

IGF1 is being tested alone in a large-scale, multicenter trial in ALS, for which data analysis is scheduled to begin this summer. That compound, known as Myotrophin, failed to meet criteria for FDA approval in two earlier trials.

Some 50 people with ALS in Italy are receiving Iplex through Insmed’s “expanded access” program. “We hope data collected from the Italian patients who are participating in this expanded access program will be useful for further clinical development of Iplex and/or IGF1 for this indication,” said Geoffrey Allan, president and CEO of Insmed.