HEMISPHERX BIO : Hemispherx Biopharma Receives Complete
05 févr. 2013•07:31
Hemispherx Biopharma Receives Complete Response Letter From FDA
PHILADELPHIA, Feb. 4, 2013 (GLOBE NEWSWIRE) -- Hemispherx Biopharma, Inc. (NYSE MKT:HEB) (the "Company" or "Hemispherx"), announced that it received a Complete Response Letter from the US Food and Drug Administration ("FDA") declining to approve its new drug application ("NDA") for Ampligen(R) for Chronic Fatigue Syndrome ("CFS"). The FDA said Hemispherx should conduct at least one additional clinical trial, complete various nonclinical studies and perform a number of data analyses.
In its Complete Response Letter ("CRL"), the FDA set forth the reasons for this action and provided recommendations to address certain of the outstanding issues. The Agency stated that the submitted data do not provide substantial evidence of efficacy of Ampligen(R) for the treatment of CFS and that the data do not provide sufficient information to determine whether the product is safe for use in CFS due to the limited size of the safety database and multiple discrepancies within the submitted data.
In the two pivotal clinical studies that form the basis of approval for Ampligen(R), Hemispherx believes that the primary efficacy endpoints were met and that they showed a statistically significant improvement (i.e., with a p-value of 0.05 or less). The FDA and Hemispherx do agree that in clinical study AMP-502, the primary endpoint was met (p=0.02). In clinical study AMP-516, the FDA's analysis resulted in a p-value of 0.10, while Hemispherx's calculation resulted in a p-value of <0.05, and yet both analyses indicate that those patients on Ampligen(R) improved over those on placebo. With regard to safety, Hemispherx has provided data from the 845 subjects who have received Ampligen(R), including 589 subjects suffering from severe CFS and over 200 CFS patients who have received Ampligen(R) for at least one year or longer. The Company believes that these data are sufficient to determine the safety profile of Ampligen(R). At the December 20, 2012 FDA Advisory Committee meeting, 8 of the 13 Advisory Committee members voted yes on the question of "Is the safety profile of Ampligen(R) adequate for approval for the treatment of CFS?"
Hemispherx plans to request an end-of-review conference with the FDA as a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug Evaluation and Research regarding the Agency's decision. The purpose of the conference is to review all of the issues raised in the Agency's CRL as well as to discuss the corroborating data and experiences of clinicians and patients who have seen the benefits of Ampligen(R) therapy.
Hemispherx has become aware that a prominent CFS advocate and long-time CFS sufferer, who has been on Ampligen(R) since 1999 through a treatment IND, began a hunger strike on January 30, 2013 to seek FDA approval of Ampligen(R). Hemispherx understands the frustration that there is still no FDA-approved treatment for CFS and the concern that patients may lose access to Ampligen(R) therapy. Out of concern for the health of the CFS community, Hemispherx has asked any hunger strikes be discontinued and that patients join in a collaborative effort between the FDA, Hemispherx, CFS clinicians and patient advocates to find a solution to this significant unmet medical need.
In the past, the FDA has shown great willingness to work with stakeholders to find solutions for serious and life-threatening illnesses. Dr. Margaret Hamburg, Commissioner of the FDA has previously stated that, "FDA has an important role to play in shaping the future of medical breakthroughs by bringing stakeholders together to identify and overcome challenges." Hemispherx hopes that the FDA will view the Ampligen(R) end-of-review conference as an opportunity to involve patient advocacy, clinicians and researchers in a concentrated effort to do something for these patients over the near-term, including further evaluation of how new legislation, such as the recently enacted "FDASIA" statute, may have a role in finding a solution. The views of one internationally recognized researcher/clinician, Dr. Nancy Klimas, who has over 20 years' experience evaluating and treating CFS patients, can be found at http://www.sciencedaily.com/releases/2013/01/130124183448.htm