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).
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.
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.
- For More on Ampligen and ME/CFS See Phoenix Rising’s Ampligen ME/CFS Resource Center
- Check out Hemispherx’s Press Release on its response to the FDA
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