Debate Over Experimental ALS Drug Heats Up

FDA logo
FDA logo
Genervon has requested accelerated approval from the FDA for its experimental ALS drug, GM604.

The FDA, along with a growing list of neurologists, is urging Genervon, the drug company behind an experimental drug for amyotrophic lateral sclerosis (ALS), to release full trial data so the disease community can make more informed decisions about future treatments.

The drug, GM604, which has garnered public and patient support via a petition, is at the center of a growing debate between medical professionals and patients, who are pushing for broad access to the drug before phase 3 trials have been conducted. Alternately, neurologists and related medical professionals have expressed varying levels of skepticism about the drug, trial results, and the privately-owned company behind it.

Genervon, owned by Dorothy and Winston Ko, released top-line results from a 12-week, phase 2 trial in 12 ALS patients back in October, but has yet to make all of the trial’s data public. “When comparing ALS patient receiving GM604 and placebo in our Phase 2A trial, the GM604 treated group achieved statistical significance not only in FVC clinical data but also in three well known ALS biomarkers,” the company said in a press release.

After the trial, the company reportedly submitted the data to the FDA in hopes of garnering accelerated approval of the drug, which would make it publically available prior to undergoing a phase 3 randomized, controlled trial to determine the drug’s efficacy.

By law, the FDA is prohibited from releasing this data to the public, placing the responsibility to publish the complete trial data on Genervon.

”We call upon Genervon to release all the data from their recently completed trial in order to allow a more informed discussion of the trial findings among ALS stakeholders,” the FDA said in a statement. “Such a release should include the pre-specified clinical outcome measures as assessed by change from baseline observations that were taken just prior to randomization to drug or placebo. Such data provide the strongest basis to assess for drug-related changes in efficacy and safety parameters.”

In the meantime, Genervon has released several related press releases, including personal responses to statements made by detractors, as well as “layman versions” of informational releases about the drug.

Do you think Genervon should receive accelerated approval for GM604? Tell us in the comments.