"The FDA Will Listen To Your Voice If You Choose To Express It"

The letter below was received by an ALS caregiver/widower/advocate. He had inquired for information from Genervon about their GM6 drug candidate.  GM6 was also in the news again this week.  There is an interesting conversation on the forum at als.net that includes the gentleman with ALS treated in the recent news item.

Please read and consider and act.  It has been two years since the FDA ALS hearing.  We have a chance to speak again on something immediately actionable. If there is one thing I hope we can especially appreciate this week, it is our freedom of expression. Thank you.

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Dear All,

Thank you for writing to Genervon about your interest in GM6. Attached are our two latest press release of our trials results. Sorry there is no giddy up option to Tijuana clinic or several variation of it as suggested by a few of you.

Genervon is meeting FDA next month for accelerated approval of GM6 for all ALS patients. Our plea is that ALS patients cannot wait and GM6 is very safe with no significant adverse events in any treatment group patients, plus incidentally GM6 is efficacious including Genervon trial programs for other neurodegenerative diseases. Genervon argued that GM6 should be advanced to conditional approval in a post-marketing surveillance program based on the following:
1. Demonstrated safety

2. Certain death for thousands of ALS patients during the time necessary for a Phase 3

3. Statistically-significant efficacy between treated and placebo groups in biomarker data

4. Statistically significant efficacy between treated and historical control groups in clinical data

5. Demonstrated ability to regulate homeostasis


As the FDA undoubtedly appreciates, an entire generation of newly diagnosed ALS patients likely would die in the time it would take Genervon to conduct another round of clinical trials before GM604 could be approved and made available to treat ALS patients. Although Genervon acknowledges the paramount importance of public safety in the FDA’s drug approval process, it also believes that ALS presents a compelling case for an exception to the usual process. This view is of course shared by many sufferers of ALS.

However, the chance of a quick approval by FDA is very small. FDA has the mandate and mindset to know as much as possible about the disease and the new drug no matter how long it would take. But all of you being an important constituent FDA will liTsten to your voice if you choose to express it. Your expression should be appropriate and appreciative. The best contact would be CDER 

http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ContactCDER/default.htm

You can also express your support through social media, ALS non-profit associations or other channels. 

A sample of a patient's voice: 
"It is horrifying to be told that you will die very quickly and along the way be reduced from a vibrant adult to less than an infantile state, all while retaining your cognition but losing the ability to express it. I live this way every day. Every waking moment is a fierce battle between iron determination and utter despair. For the first time in history we have a treatment with major effect and an excellent safety profile. PALS need treatment options RIGHT NOW. The risk of GM6 at this point is extremely acceptable to us, especially given that without treatment the risk is 100% fatal. We cannot wait another few more years while tens of thousands of us die. Please allow GM6 preliminary approval and market surveillance for final determination. Doing so would very likely mean rescue from ALS for many tens of thousands and certain hope for all."

INFO@genervon.com
Genervon Biopharmaceuticals LLC
1055 E. Colorado Blvd. Suite 500
Pasadena, CA 91106
Tel: (323)-721-5500
Fax: (323)-721-9131

This email is sent on behalf of Genervon Biopharmaceuticals, LLC. It may be privileged and contains confidential information intended only for the use of the recipient(s) named above. Use by anyone else is strictly prohibited. If you have received this communication in error, please notify us immediately. 

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