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FDA Asks Genervon to Release Complete Data from Controversial ALS Study

The US Food and Drug Administration (FDA) issued a statement calling on Genervon Biopharmaceuticals to release complete data from their recent trial evaluating an experimental drug for treatment of amyotrophic lateral sclerosis (ALS).

The agency is prohibited by law from releasing confidential information about experimental drugs, however the agency requested that Genervon provide full data from the trial evaluating GM604 to allow a more thorough discussion of study findings from the ALS stakeholders.

ALS is a rare and debilitating neurological disease, which currently has no cure. The disease affects nerve cells in the brain and spinal cord. Early symptoms of the disease often include increasing muscle weakness, particularly in the arms and legs, speech, swallowing or breathing. The progressive degeneration of motor neurons eventually leads to death. The average life expectancy for ALS patients is 2-5 years. While more than 160 clinical trials in ALS have been conducted by over 60 companies, all have failed except for riluzole which is not curative and provides modest effect of increase survival, but no improvement in quality of life.

Last October, the company released clinical trial results from a Phase IIa clinical trial, enrolling 12 patients, with eight receiving GM604 and four receiving placebo. The study assessed multiple biomarkers for the disease, as well as an impact on ALSFRS-R rates and forced vital capacity (FVC). Genervon has yet to release ALSFRS-R data, however the company did reveal a major improvement in rate of decline for FVC compared to placebo.

Additionally, the company provided details from a compassionate use case, in which the patient experienced a rebound from the disease. The patient had improvement in swallowing capacity.

Following these results, Genervon applied for accelerated approval from the FDA, which provides promising treatments for serious or life-threatening diseases the ability to bypass large-scale efficacy trials, allowing the drug to enter the market quicker. These results and Genervon’s claims resulted in an influx of responses from ALS patients pleading to grant the approval.

It is not often that drugmakers request accelerated approval for results from an only 12-person trial. Scientists say that it is impossible to measure a drug’s effect with minimal data and shortcutting the traditional clinical trial process could lead to issues down the line.

A few weeks ago, Genervon released a statement to ALS patients and caregivers saying that if the FDA did not provide accelerated approval and the company is required to conduct a Phase III trial, it would take at least three years for the drug to reach NDA stage, and said, “This means the majority of this generation of ALS patients would not survive to try GM604.”

The FDA responded asking Genervon to release all of the data from the completed trial, such as pre-specified clinical outcome measures assessed by change from baseline observations that were taken just prior to randomization to drug or placebo. The agency claimed that such data would provide the strongest basis to assess for drug-related changes surrounding safety and efficacy.

Source: Food and Drug Administration; Genervon Biopharmaceuticals

Last updated: 4/17/15; 1:55pm EST

 

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