Insmed's Iplex drug

[Sparty2]

INSM FDA announces position on allowing patients with ALS access to IPLEX under an IND (0.98 +0.08)
The FDA decided to allow patients with Amyotrophic Lateral Sclerosis, a fatal neurodegenerative disease also known as Lou Gehrig’s Disease, or ALS, to have access to Insmed's (INSM) Iplex under an Investigational New Drug (IND) application. The FDA and Insmed have agreed that access to Iplex for investigational use in patients with ALS will occur in two ways under Investigational New Drug applications (INDs):
Single-patient INDs requesting “compassionate use” of Iplex for treatment of named patients with ALS, received and date-stamped by FDA’s document room by close of business on March 6, 2009, will be allowed to proceed, and Insmed has agreed to supply Iplex to those patients; and
The remaining supply of Iplex, which is very limited, will be used by Insmed to conduct a clinical trial under an IND in which other patients with ALS who are interested in receiving Iplex treatment will be randomly assigned to receive drug through a lottery system.

 

[Al]

Iplex and ALS

From ALSA Bulletin.

FDA, Insmed, Inc. Announce Special Program for IPLEX and ALS

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/infopage/mecasermin_rinfabate/default.htm.

For additional information from The ALS Association, please contact our Patient Services Department at [email protected].

 

[nishant]

Not to sound negative, but I asked my neuro about this in Jan. He told me that doctors at Mayo have done research on the compound this drug uses and haven't found any beneficial effects of it on ALS patients.

 

[Al]

My Neuro also thinks it will end up like Lithium. A flash in the pan, but no substance when subjected to intense scrutiny.

AL.

 

[rocmg]

our neuro didn't seem to think too much of this either... she sent me a report called "iplex and the telephone game." if anyone is interested in reading the document, private message me your email and i will forward it.

 

[Al]

Here's the link to a short discussion we had on the telephone game.

AL.

https://www.alsforums.com/forum/showthread.php?t=7424&highlight=iplex+telephone+game