In recent months, Pasadena-based Genervon has galvanized many patients with ALS by repeatedly touting the results of 12-week, 12-person trial involving the company's drug, GM604. The company asserted its early results were “statistically significant,” “very robust” and “dramatic.”
It also has said it "submitted an accelerated approval application" to the FDA which, if approved, "would allow immediate access" to patients with ALS, also known as Lou Gehrig's disease.
But the Wall Street Journal reported Monday that Genervon said in an email that it is “at the point of communicating with FDA about whether [the agency] would accept our formal application” for accelerated approval. In other words, the company has not yet submitted a New Drug Application, a step needed to officially set the FDA approval process in motion.
The company's acknowledgement that it has not filed an NDA appears to contradict earlier press releases and statements made by the firm's owners, Winston and Dorothy Ko -- or at least to have sown confusion about the actual status of GM604. In one February press release, for example, the company said that in a meeting with the FDA, "three times during the one-hour meeting we requested that the FDA grant GM604 accelerated approval."
Asking, however, is not the same as filing the necessary paperwork and the accompanying data required for the FDA to accept it as sufficient. The difference might seem to be a matter of semantics. But the real-world consequence is that, if Genervon has no application pending at the FDA, there is no imminent decision for the FDA to make about approving GM604.
That would be a disappointment to ALS patients who, encouraged by statements by Genervon's owners and eager for a treatment for a devastating disease with no cure, have helped fuel a Change.org petition pressing the FDA to rapidly approve the drug. The petition has 536,000 signatures and counting. Supporters held a rally in Washington last month, and sought to enlist the help of lawmakers on Capitol Hill. Another rally has been scheduled for May.
Last week, the FDA took the unusual step of publicly calling 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." In a statement, the company said it had “submitted all raw data, analysis and reports” to the agency.
The Washington Post last week asked the company to clarify whether it had submitted a formal application to the agency, but the company did not answer the question in a brief reply.
On Tuesday, the Post asked again. This time, Genervon replied with a statement saying company officials had "filed a formal request for the Accelerated Approval Program and are now waiting for a final decision. That formal request was made February 24th with supporting documents." The company acknowledged that it hasn't filed an NDA, but reiterated that it is communicating with the FDA about whether the agency "would accept our formal application for GM604 with approval based on surrogate endpoints under the Accelerated Approval Program. This is a typical step in the process."
The company added, "Some are crating [sic] an issue out of nothing hoping to discredit Genervon and causing delay to make treatment available to ALS sufferers."
Traditionally, even after a company submits a formal application to the FDA, the agency has 60 days to decide whether to formally accept it. When an application is accepted, the agency would have six months or longer to review the merits of the drug before deciding whether to make it widely available, meaning that GM604 is unlikely to reach patients in the near term. An FDA spokeswoman said Tuesday the agency is prohibited by law from discussing any materials submitted by a company developing a new drug.
The confusion over the status of Genervon's application has done little to quell the ongoing debate over how to interpret the results of the small, early trial for GM604. Many ALS patients remain hopeful, and argue the FDA should grant access to the experimental treatment, given the severity of the disease and the fact they are willing to accept any risks.
But plenty of skepticism remains about the drug, and some experts worry that Genervon has unfairly stirred false hope among ALS patients.
Steve Perrin, president and chief scientific officer of the ALS Therapy Development Institute, a nonprofit dedicated to developing effective treatments, told the Post weeks ago that "there is no mathematical way or statistical way that they could measure a drug effect” in such a small trial. He followed with a detailed blog post recently, concluding that "GM604 has not been adequately tested in patients."
The ALS Association likewise has declined to push for approval for GM604, saying that making the drug widely available could expose patients to possible side effects and “pulls money, personnel and effort away from finding the cure that all of us are working together to find.”