FDA Pushes Genervon to Release All Trial Data on ALS Drug
The FDA is taking the unusual step of pressing Genervon Biopharmaceuticals to release all data from a Phase IIa trial of its amyotrophic lateral sclerosis drug, including an analysis of changes in patients’ lung function.
The FDA says it is prepared to use all expedited development and approval pathways available to further mutual goals of developing treatments to battle ALS.
Because Genervon hasn’t published the data, some of which could be proprietary, the FDA can’t require the drugmaker to make it public. Genervon said last week it is considering the request.
Patients and their families began pressing for the experimental therapy after Genervon announced that its GM604 drug helped to slow the decline in ALS patients’ ability to breathe.
Patients pointed out that life expectancy post-diagnosis is between two and five years. The only available drug, Sanofi’s Riluzole (rilutek), extends life by only a few months, and GM604 would offer another option, they say. The push to make GM604 available to a wider group of patients has been controversial, however, since Genervon’s trial involved only 12 patients.
Genervon says it has had contact with the FDA throughout the trial process and received a positive nod on its primary endpoints. The firm met with FDA staff in February to seek accelerated approval of the drug, a decision that is pending. — Lena Freund