July 28, 2009 

INSMED To Limit Supply of IPLEX to Existing
ALS Patients in Current Program

INSMED, the manufacturer of IPLEX, has announced that effective immediately, it will limit the supply of the drug to the 70 ALS patients receiving the drug in the United States and other parts of the world as part of a study to determine its safety and therapeutic potential.  The biopharmaceutical company also announced that it will not initiate further clinical trials with IPLEX at this time.  There are 12 people in the United States currently receiving IPLEX as part of an agreement between the U.S. Food and Drug Administration and INSMED.

The Richmond-VA.-based INSMED also announced that it no longer has the capability to manufacture IPLEX, a drug that combines insulin-like growth factor (IGF-1) and IGF binding protein 3.  IPLEX has not been approved for use in treating ALS.  The company said any agreement with another company to produce the drug would not result in production of additional quantities for at least 12 to 18 months.

The Association encourages the FDA and INSMED to establish partnerships with the ALS community to ensure that the program yields meaningful results that will guide the next steps in determining whether IPLEX is effective and safe for people with ALS.

Based on existing clinical and scientific evidence, The ALS Association cannot encourage or recommend the off-label use of this medication without substantive evidence of its efficacy through a rigorous clinical trial.  The ALS Association is continuing to monitor and assess information about IPLEX as it becomes available to provide the public with the most up-to-date reports about its potential for ALS.

According to INSMED, it has sufficient inventory of IPLEX to supply the 70 patients worldwide who are being receiving the drug for ALS.  The patients in the United States are receiving IPLEX under single-patient Investigational New Drug applications approved by the FDA for 24 months. 

IPLEX originally was approved in the United States as a treatment for children with growth failure, but it is now discontinued and no longer available for this population.  The drug was utilized in a now-closed study involving myotonic muscular dystrophy (MMD).  INSMED is supplying IPLEX to the Italian government under an “extended access program,” but it continues to be an untested and unproven treatment for ALS in the United States.

The company said it plans to analyze the data being received from the ALS and MMD programs and assess the overall development program for IPLEX, including possible manufacturing options and future clinical trials.  Initiation of the Phase II clinical trial for ALS patients in the U.S. that was discussed with the FDA earlier in 2009 has been postponed while INSMED conducts the assessment, according to the company.

For additional information from The ALS Association, please contact our Patient Services Department at [email protected] or toll-free at 1-800-782-4747.