FDA to Genervon: Do the right thing ... or else.

  • by: |
  • 04/22/2015

It looks like the FDA (and specifically John Jenkins) has had enough and isn't going to take it anymore.

From the pages of Medical Marketing & Media …

FDA takes unusual stance on ALS drug

The FDA said Genervon should supply more data if it wants an accelerated approval.

The FDA took an unusual public stand Friday when it publicly asked Genervon Biopharmaceuticals to release all data from the California drugmaker's recently completed trial for an experimental drug for amyotrophic lateral sclerosis, a neurodegenerative disease also referred to as ALS and Lou Gehrig's disease.

Doing this would allow a more informed discussion of the trial findings among ALS stakeholders for GM604, the FDA said.

“It is certainly not a routine practice,” FDA spokesperson Sandy Walsh told MM&M.

The FDA's request follows a surge of patient activism, including a Change.org petition that is urging the FDA to grant an accelerated approval to the drug based on a Phase-IIa study comprising 12 patients. An accelerated approval would allow the drug to be distributed as is to the general patient population as opposed to using pathways like the FDA's compassionate use provision, which grants patient-by-patient access to unapproved investigational drugs.

The FDA has a history of taking patient lobbying efforts into consideration, such as when it approved Sanofi's Lemtrada for multiple sclerosis.

Steven Perrin, CEO of the ALS Therapy Development Institute, which conducts ALS research, highlighted what he considers problems with the research on his blog. He told MM&M that the data set is far too small for a regulatory decision for reasons that include the size of the clinical trial and because ALS progression differs patient by patient. He said this makes it difficult to back efficacy claims.

Perrin said putting ALS patients on a drug lacking sufficient data could jeopardize other experimental drugs that do have Phase-III data because patients taking GM604 would not be able to take another candidate, be it one that his group supports or one that is backed by another company.

Perrin said he doesn't want to kill the drug but he does want Genervon to “do the right thing” and adhere to traditional regulatory protocols and run a larger clinical trial.

Perrin is not Genervon's only critic. The Washington Post reported earlier this month that one of the researchers involved in the study said more data was needed.

Genervon said in press releases that finding a treatment for the disease requires an unconventional approach. Genervon declined to provide answers to queries including if the company plans to pursue late-stage clinical trials and if it has provided the FDA with all of the information it has on hand. Chief Operating Officer Dorothy Ko referred MM&M to its website.

Genervon said in an April 13 press release that pursuing an accelerated approval has some risks because it would mean exposing this early-stage treatment to “a full spectrum of heterogeneous ALS patients,” rather than if the drug went through traditional testing, which the company described as “a more secure and conservative approach.” Genervon also said drug sales from an accelerated approval would allow the company to fund further development.

The FDA noted in its public request that it is not allowed to release clinical trial data about experimental medications, but the drugmaker can.

Perrin acknowledged that it is easy to understand the appeal of an experimental medication but that choosing a drug that is not scientifically supported can prevent patients from trying one that has more verifiable outcomes. “They are desperate for hope and they want access to stuff, even if there is not a lot of data,” he said.

Genervon told MM&M it is reviewing the FDA's request.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

Blog Roll

Alliance for Patient Access Alternative Health Practice
Better Health
Biotech Blog
CA Medicine man
Cafe Pharma
Campaign for Modern Medicines
Carlat Psychiatry Blog
Clinical Psychology and Psychiatry: A Closer Look
Conservative's Forum
Club For Growth
Diabetes Mine
Disruptive Women
Doctors For Patient Care
Dr. Gov
Drug Channels
DTC Perspectives
Envisioning 2.0
FDA Law Blog
Fierce Pharma
Fresh Air Fund
Furious Seasons
Gel Health News
Hands Off My Health
Health Business Blog
Health Care BS
Health Care for All
Healthy Skepticism
Hooked: Ethics, Medicine, and Pharma
Hugh Hewitt
In the Pipeline
In Vivo
Internet Drug News
Jaz'd Healthcare
Jaz'd Pharmaceutical Industry
Jim Edwards' NRx
Kaus Files
Laffer Health Care Report
Little Green Footballs
Med Buzz
Media Research Center
More than Medicine
National Review
Neuroethics & Law
Nurses For Reform
Nurses For Reform Blog
Opinion Journal
Orange Book
Peter Rost
Pharm Aid
Pharma Blog Review
Pharma Blogsphere
Pharma Marketing Blog
Pharmacology Corner
Pharmaceutical Business Review
Piper Report
Prescription for a Cure
Public Plan Facts
Real Clear Politics
Shark Report
Shearlings Got Plowed
Taking Back America
Terra Sigillata
The Cycle
The Catalyst
The Lonely Conservative
Town Hall
Washington Monthly
World of DTC Marketing
WSJ Health Blog