Below is the FDA's response to my email encouraging the FDA to approve Ampligen. Their response is a PR masterpiece. If the FDA, NIH, and CDC spent as much money on researching ME/CFS as they do on their Public Relations efforts, we would have a cure by now.
Dear Erica Verrillo,
Thank you for sharing your personal testimony as
well as your desire for the U.S. Food and Drug Administration (FDA) to approve
Ampligen. Please accept this response on behalf of FDA’s leadership, who
forwarded your email to the Division of Drug Information for direct
reply.
As evidenced by the hundreds of letters, emails, and testimonies
submitted to FDA, Myalgic Encephalomyelitis (ME)/Chronic Fatigue Syndrome (CFS)
is a devastating disease with a serious impact on quality of life. We are
acutely aware of the seriousness of this disease, that no FDA approved
treatments are available, and of the community’s strong desire to see
rintatolimod injection (Ampligen) approved.
For many years FDA has worked
with Hemispherx Biopharma, Inc. (Hemispherx) on an approval pathway for
Ampligen. Since the time of the original New Drug Application (NDA) submitted by
Hemispherx for the use of Ampligen to treat CFS in 2007, FDA’s review division
has provided many specific recommendations on how best to address deficiencies
in the application. In 2009, the review division asked Hemispherx to conduct at
least one additional controlled clinical study showing a convincing effect in
the CFS population. No new trials were conducted. Hemispherx conducted
additional analyses of their existing data, which FDA agreed to review in a
resubmitted NDA.
A public meeting of FDA’s Arthritis Advisory Committee
(AC) on December 20, 2012, was held to provide FDA with independent scientific
and clinical expertise regarding the Ampligen NDA. At the meeting, both
Hemispherx and FDA reviewers presented assessments and analyses of the NDA data
to the experts, including physicians with expertise in CFS, a CFS patient
representative, and an industry representative. The majority of AC members were
concerned about the lack of consistency within the clinical trial results, as
well as the limited size of the database available to evaluate Ampligen. The
members shared FDA’s concerns, as well, about how the studies had been
conducted, including multiple discrepancies and gaps in the safety data. At the
end of a full day of discussion AC members voted 8-5 against the approval of
Ampligen for the treatment of patients with CFS because of insufficient safety
and efficacy data.
On Monday, February 4, 2013, Hemispherx announced the
receipt of a Complete Response (CR) letter from the FDA for Ampligen. FDA issues
a CR letter to convey that our review of an application is complete and we
cannot approve the application in its present form. A CR letter describes all of
the specific deficiencies that the Agency has identified in an application,
allowing the company an opportunity to correct those clearly defined
deficiencies in a re-submission. FDA’s decision regarding Ampligen encompassed
many factors, including the safety and efficacy data and the advice of the AC.
We understand the frustration and pain of ME/CFS patients and their caregivers,
and how important it is that we continue to work toward development of
treatments.
We want to emphasize that the CR letter issued for Ampligen
is entirely separate and distinct from FDA’s support of drug development
pathways for CFS – these initiatives remain unaffected and fully supported.
ME/CFS is a serious disease and treatments for it represent an important area of
unmet need. We will continue to encourage the pharmaceutical industry to
develop new treatments in this area. To assist companies with their development,
FDA is sponsoring a workshop in spring 2013 focused specifically on ME/CFS drug
development.
We express our gratitude to you and the ME/CFS community for
your unwavering support of the research and care of those with ME/CFS. We join
you in this commitment, and we look forward to exploring how best to facilitate
and expedite the development of safe and effective drug therapies for the signs
and symptoms of this debilitating disease.
Best regards,
Mary
Kremzner, PharmD
Director, Division of Drug Information
Center for Drug
Evaluation and Research
Food and Drug Administration
For up-to-date
drug information, follow the FDA's Division of Drug Information on Twitter at
http://twitter.com/FDA_Drug_Info
This communication is
consistent with 21CFR10.85(k) and constitutes an informal communication that
represents our best judgment at this time but does not constitute an advisory
opinion, does not necessarily represent the formal position of the FDA, and does
not bind or otherwise obligate or commit the agency to the views expressed.
