FLICKR, SLGCKGCLast fall, California-based biotech Genervon announced results of a Phase 2 trial involving 12 amyotrophic lateral sclerosis (ALS) patients: its drug candidate, GM604, had produced “very robust” and “dramatic” results, according to company news releases. In February, the company applied to the US Food and Drug Administration (FDA) for accelerated approval, which would allow the drug to skip Phase 3 and head straight to market. Patients, who typically only live two to five years past diagnosis and have no effective treatment options, quickly began advocating for the FDA to grant the company’s request, but many researchers believe that a larger trial is necessary before the drug becomes widely available, The Washington Post reported last week (April 3).
“All this petitioning and press releases over such little data is premature,” David Gortler, a pharmacology expert and former FDA senior medical officer, told The Washington Post. “I think Genervon is preying on the lack of information that the average person has about the drug-development process. . . . You can’t rush the scientific process. Good science takes time.”
But time is not something that most ALS patients have at their disposal, and advocates for GM604’s accelerated approval argue that, if the drug is forced to continue down the traditional clinical trial path, the majority of today’s patients will have died before it reaches the market. Patients and advocates have thus petitioned for its rapid approval, collecting more than half a million signatures on Change.org ...
