According to a report in FierceBiotech …
The Wall Street Journal has caught up with the Genervon controversy, recapping a series of events from the last few days that followed the big social media campaign to gain an instant approval for a new ALS drug based on the results of a 12-patient study. FierceBiotech readers will know already that the data for GM604 were questioned by Steve Perrin, president and chief scientific officer of the ALS Therapy Development Institute, and that the FDA followed up a day later with an extraordinary challenge to Genervon to publish the data on a drug that the biotech has touted as a major advance for patients.
The Journal, though, did manage to nail down an answer from the company, run by Winston Ko, about the application the patient community believes has been filed for accelerated approval.
There isn't one.
Genervon tends to prefer email communications these days. And the company told the Journal that it is "at the point of communicating with FDA about whether [the agency] would accept our formal application" for accelerated approval.
Genervon, meanwhile, has already repeated claims to us that it made a "formal request" for accelerated approval, an assertion that dates back at least to last month. The company has stated in releases that it has "met with the FDA and has submitted an Accelerated Approval application." The biotech has also claimed several times that an accelerated approval decision was already in the works at the FDA, and it's done nothing to stop publications like The Washington Post from reporting that an application is at the FDA as it repeatedly prodded dying patients and their families to back calls for a quick approval as their only chance of getting the drug and staying alive.
Back in mid-February, Genervon issued a statement saying that it had met with the FDA and asked for accelerated approval three times during the course of the get-together, then asserted that the agency had 30 to 60 days to respond. That mid-April deadline--which has been repeated by the petition's sponsors--helped trigger a frenzy of petition signing at change.org, which currently has more than 535,000 signatures backing instant approval.
But that's not going to happen, because without an actual application, there is no 60-day response time for the agency to accept or reject the application for review. Given the FDA's challenge to Genervon, and its clear doubts about the data from the tiny study, the company's chances of being received at a wary FDA with open arms lies somewhere between none and zero.
As far as making any public statements about Genervon, though, the agency's hands are tied by federal law.
Patients and their families should know that there is no decision pending at the FDA. They might also ask themselves if Winston Ko and Genervon have been stirring false hope among the dying in an attempt to create a social media campaign that would push the FDA to sanction the drug's sale on questionable data.
