The potential chronic fatigue syndrome (ME/CFS) drug Ampligen (rintatolimod) is about to go up for FDA approval again, but are its chances any better this time? It has elements both for and against it.
Three years ago, the FDA gave Ampligen's manufacturer, Hemispherx Biopharma Inc., a "not now" response in what's called a Complete Response Letter, requesting more data to prove the drug's effectiveness. Hemispherx submitted more data, and the new decision is due in just over a month.
However, it's not the data that makes some Ampligen watchers think the drug will make it through this time - it's a changing political climate surrounding ME/CFS.
Some financial blogs, which have been skeptical about Ampligen until recently, now say they think approval is highly likely. Here's a quote from investment blog Seeking Alpha:
"Patient advocacy groups, U.S. Senators, and the U.S President have all pushed for an FDA approved therapeutic option for ME. Let's be clear on this: there is no alternative to Ampligen at this time nor will there be any time soon. Hence, this could be the last chance to get an ME therapy approved for several years. It's also important to note that upon accepting Hemispherx's New Drug Application last July, the FDA agreed that the review will focus on the drug's efficacy, not safety. So the FDA does feel comfortable with the drug's safety profile, and this will have a major impact on the final outcome." -Seeking Alpha, Dec. 12, 2012
The FDA, at an ME/CFS stakeholder meeting in September, talked about the possibility of getting a treatment approved quickly:
"FDA's committed to making promising drugs available to individuals with serious diseases as quickly as possible for the rapid development and review of these types of therapy. For example, you may have heard the term, 'the accelerated approval process.' This is in FDA's regulations...this process is set up to increase the availability of experimental drugs and biological products based on findings that are somewhat preliminary, but thought to predict clinical benefit." -Transcript, FDA ME/CFS Stakeholder Meeting, Sept. 13, 2012
To qualify for accelerated approval, the FDA requires that the drug:
- Treats a serious or life-threatening disease (which the FDA now admits ME/CFS is);
- Provides meaningful and measurable therapeutic benefit to patients over existing treatment.
That last bit is really important, because currently ME/CFS has no existing FDA-approved treatment. Any proven benefit to any number of people could be considered meaningful and measurable.
However, the talk of a rosier outlook came before the Dec. 20 meeting of the FDA's Advisory Committee. That committee gives recommendations to the agency, and the agency usually follows them - but it has no obligation to do so. The committee voted 8-5 against Ampligen. It's important to remember, though, that the committee also recommended against Lyrica (pregabalin) for fibromyalgia, and it was approved. Why? Because, at the time, no other drugs were approved for fibromyalgia. So will the committee's vote mean anything?
We won't have to wait long to find out. The FDA's decision is due Feb. 2, 2013.
Do you think some of these factors will help Ampligen become approved? Is it dead in the water? If Ampligen were available and affordable, would you try it? Leave your comments below!