FDA, Insmed, Inc. Announce
Special Program for IPLEX



This morning, the U.S. Food & Drug Administration granted approval for a limited number of people with ALS in the United States to receive IPLEX, a drug that combines insulin-like growth factor (IGF-1) and IGF binding protein 3. IPLEX, which has not been approved for use in ALS, is manufactured by Richmond, VA-based Insmed, Inc. The new program is the result of an agreement between the FDA and Insmed.

The ALS Association hopes that the FDA-approved program will develop informative data about IPLEX that can lead to a better understanding of its efficacy and safety and enable both patients and clinicians to make more informed decisions about the use of IPLEX and its potential as a therapy for ALS. To this end, 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 ALS.

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 is being tested in a now-closed study involving myotonic muscular dystrophy (MMD). Insmed is supplying IPLEX to the Italian government under an "expanded access program," but it continues to be an untested and unproven treatment for ALS in the United States.

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.

Additional information on today's announcement is available here: http://www.fda.gov/cder/drug/infopag...te/default.htm.

For additional information from The ALS Association, please contact our Patient Services Department at alsinfo@alsa-national.org or toll-free at 1-800-782-4747.





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