FDA takes unusual stance on ALS drug

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