Dear supporters of Hope NOW for ALS,
Please email Congress before Friday to ask for their continued support. Due to the upcoming recess, this week is our only opportunity to request their support before our next teleconference with the FDA on July 6.
On June 17, we conducted a teleconference with the FDA and we can report some progress, but also some concerns.
The good news is the FDA agrees that:
1) FVC is a valid clinical endpoint,
2) trials can be populated with “fast progressors,” and
3) an ALS therapy could be approved after a Phase II study if it resulted in a clinically significant, positive effect on FVC decline.
FDA described what they meant by clinically significant as “compelling, convincing evidence” between the treated group and the control (placebo) group.
The FDA’s position on potential ALS biomarkers is that none have been validated and, therefore, none can be used as surrogates to support an accelerated approval, but they did assure us that collecting data on potential biomarkers would not be used as evidence against approval of an ALS drug if a correlation between the marker and the primary endpoint (for example FVC) is not seen. Also, FDA agreed that measurement of potential biomarkers could be included in a study to enhance understanding of their relationship to ALS. FDA provided some general responses on how an ALS biomarker could be validated, but did not progress to details.
We proposed the concept that small clinical studies can produce sufficient evidence of efficacy to support an approval, especially if progression prediction models are employed to appropriately select the trial population or to establish individual patient baselines. The FDA expressed openness to learning more about predictive algorithms.
During discussions of some of the potential biomarkers for ALS, it appeared that the FDA’s Division of Neurology Products (DNP) medical review officers (Drs. Eric Bastings and Ronald Farkas) were unfamiliar with the state of biomarker science in ALS. We hope this is not the case, and we intend to explore and build their knowledge of the state of medical science with respect to ALS during our ongoing communications.
Based on our discussions during the meeting, we are also concerned that the FDA is employing a very narrow, rigid and outdated view of the circumstances in which the Accelerated Approval pathway should be available to drug companies trying to bring new medicines to market. Dr. Woodcock made the FDA’s position on Accelerated Approval very clear by stating that in FDA’s mind there is nothing fast about Accelerated Approval, and they wish they hadn’t called it that. Her position on the Congressional intent of Accelerated Approval seems to be at odds with the directions FDA received from Congress in the 2012 FDASIA legislation, which made it clear that FDA is to apply new thinking, flexibility and an expanded set of evidentiary tools to speed new medicines to patients who need them for diseases like ALS.
This issue will be discussed again at the next meeting since Accelerated Approval is clearly intended for diseases like ALS for which there is no effective treatment.
We are also concerned with the subjectivity the FDA maintains in determining what constitutes efficacy and “convincing evidence.” We intend to press the FDA for a clear definition of efficacy for a given trial design that could result in quickly accepting a new drug application (NDA). This is particularly relevant to GM604 in order to define a small, confirmatory study that could result in its approval if the results meet the definition of efficacy. We also believe this will create a template for future ALS treatment options.
As mentioned earlier in this update our next conference call is scheduled on July 6 and this call should determine if the Dr. Woodcock and the FDA will bend and be reasonable. Using the template below please email your congress before Friday and request their continued support.
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We’re in this together,
Jehad Majed (firstname.lastname@example.org), Matthew Bellina, Nick Grillo (email@example.com), and the Torrino Family