In April 2015, Digital Journal reported a Biotech company called Genervon had requested an accelerated approval for its experimental amyotrophic lateral sclerosis (ALS) drug after a small but promising Phase 2 clinical trial. In the article we noted several ALS suffers and campaign groups were pushing for the drugs released, while some scientists were urging caution because the drug had not been fully assessed for safety and efficacy. Now the U.S. FDA has stepped into the debate.
The completed trial has proved to be successful; however, the trial is limited since it only involved 12 people. Partly due to the campaign for the drug’s release, the manufacturer Genervon has applied to the FDA for an accelerated release. If approved, this means that a more in-depth Phase 3 clinical trial would be skipped.
The FDA has not said anything publicly about the drug, coded GM604. However, the federal agency has called on the company to publicly release all data from the Phase 2 study (at present only a summary of the trial results has been made available.)
Here FDA states: “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. 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.”
However, some people continue to remain wary. Steve Perrin, president and chief scientific officer of the ALS Therapy Development Institute, wrote a blog post where he argued that the supposedly breakthrough results achieved in the Genervon trial were not as exciting as they may seem.
Meanwhile, discussions continue and no decision is imminent about the release of the drug.
