Fast Track

 This was sent to me by another gal in the trenches.  They are trying to fast track some drugs to help us.

 

 

Dear ALS patients and Caretakers,

We are in communication with FDA after our February meeting and we have filed formal request for Accelerated Approval Program. We are still waiting for the final result. It is illegal to access GM604 without FDA approval. Genervon cannot afford the compassionate or expanded access. Phase 3 trials are very restrictive in enrollment, with limited number of patients and 1/3 of patients are randomized to placebo treatment.  The best option for the dying ALS patients in our opinion is for FDA to grant accelerated approval with post marketing Phase 4 requirements, instead of waiting for Phase 3 results which will be too late for this generation of ALS patients. All ALS patients can have access legally now and the health insurance will pay for the treatment.  However FDA has the authority to decide and the chance of approval is small.

GM604 met the AA criteria of FDA ( http://youtu.be/fzlePvW-Dg4) with FVC as the surrogate end point. Clinical end point in forced vital capacity (FVC) is an indicator of clinical survival and disease progression. Most ALS deaths are due to a decline in pulmonary involvement function. There are statistically significant differences in the change and percentage change between the treated and placebo group from screening to week 12 for all patients in FVC. The fact that these p-values are significant with so few patients is remarkable. (see Statistical Report Addendum table 3, page 9 below)
This clinical data correlated very well in terms of achieving statistical significant between treated and placebo groups with the three important ALS biomarker data TDP43, SOD1 and Tau in the excerpt of a Asahi TV ALS Special showing a GM604 treated patient. Asahi TV has the copyright of the video and it gave Genervon the right to private showing and pass along but not allowed to be posted on the internet. Please honor our promise and if you cannot keep your promise not to post it on the internet please delete the link. ( https://drive.google.com/file/d/0B-0D7Rnc4pwbem8ybVJLNUxPQkE/view?usp=sharing). The issue of small trial has been dealt with by statistical expert:   http://www.genervon.com/genervon/images/data20150203.pdf  

Links below may answer some of your questions. Please read all links carefully to minimize repeat of asked and answered questions.

http://www.genervon.com/genervon/medicines_trialdata.php

http://www.genervon.com/genervon/about_pressreleases.php