Ampligen On the Clock: Hemispherx’s ‘Complete Response’ Means Ball is Now in FDA’s Court Now

Posted by Cort Johnson

Hemispherx's Ampligen Response to FDA means the clock is ticking for the drug
By end the January, 2013, at the latest,we will know if the FDA will approve Ampligen for ME/CFS...

Moving quickly, Ampligen’s producer, Hemispherx Biopharma,  filed its ‘complete response’ to the FDA’s request for data just 53 days after the agency agreed to relax its requirements for review of the drug.  Hemispherx’s ‘complete response’ means we will know by the end of January and perhaps sooner whether Ampligen becomes the first FDA approved drug for chronic fatigue syndrome (ME/CFS).

New Data

In its response Hemispherx provided new data derived from a 24 week extension period occurring after the 40 week original trial.  Hemispherx stated the data will show that patients who remained on the drug for a further 24 weeks showed further improvements in endurance (23% – 39% increase in treadmill duration) suggesting that the drug’s effectiveness increases over time.

Similarly, Hemipherx reported that patients who responded well to Ampligen showed significantly greater improvement relative to non-responders in areas such as vitality, general health, Karnofsky performance (functionality) and activities of daily living  the longer they were on the drug.   This is important because the original study did not highlight improvements in functionality and well-being. This data suggests Ampligen works ‘globally’ to improve both endurance and functionality.

FDA and Ampligen

It appears that the FDA has at least several possibilities;

  • it can approve the drug
  • it can reject the drug
  • it can give the drug ‘accelerated approval’ status – in this scenario Ampligen is not approved but  Hemispherx is allowed to market the drug and gather more data for a final attempt at approval
  • it can ask Hemispherx for more data – without the income derived from ‘accelerated approval’ this outcome would be difficult for Hemispherx.
Hemispherx’s Press Release suggests the company is gunning for accelerated approval status. While the FDA did recently substantially relax its requirements to consider Ampligen for approval its possible, perhaps even probable, they still want that large, multi-dose, placebo-controlled, double-blinded treatment trial originally required in their 2009 report. Hemispherx didn’t come anywhere near providing that kind of data but getting accelerated approval would give them the means to do so.
 The FDA has increasingly come under fire  to approve more drugs and, in particular, to approve more drugs for chronic illnesses and  the new law provided drug companies an opening to do just that.

New Law Presents Opportunity for Hemispherx and Ampligen

In their press release Hemispherx highlighted the fact that the 2012 Food and Drug Safety and Innovation Act (FDSIA)  law encourages the FDA to take ‘innovative and flexible approaches’ to assessing treatment for ‘serious’ disorders with ‘unmet’ needs.

In section 901 from the FDASIA Act below note the emphases on ‘expedited…review’ and the need to target subpopulations.

“the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions… This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation

Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions.

Indeed, recent history suggests a drug need be effective in only a small group of patients if if few treatment options are present.  With FDA analyses suggesting that 11 lupus patients needed to be  treated on Benylstra to find one the drug helped, and with the drug showing marginal effectiveness overall, but with the last FDA approved drug for lupus dating back 50 years, Benylstra was easily approved. (Benlystra significant helped those few patients which responded.)

Hemispherx’s production facility at New Brunswick will be undergoing ‘pre-approval inspection’ as part of the FDA review process. If the drug is approved or granted accelerated approval status Hemispherx should, hopefully, be able to hit the ground running.  Could the drug be widely available to ME/CFS patients sometime next year? Time will tell.

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