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Ampligen-aanvraag FDA

(voorlopig) afgewezen:


aanvullende studie vereist.






En weer wordt de goedkeuring van Ampligen uitgesteld... (afgewezen?)


Hemispherx, de producent, wordt gevraagd

een aanvullende studie uit te voeren naar de effectiviteit van Ampligen.


Gezien de stortvloed aan geneesmiddelen die wel toegelaten worden (soms zeer versneld)

en die later weer van de markt gehaald worden en/of nauwelijks werken, en

de studies in het nabije en verre verleden die aantonen dat

Ampligen bij (een deel van de) ME/CVS-patiŽnten substantiŽle verbeteringen oplevert,

wekt de gang van zaken op zijn minst mijn verbazing.


O ja, ik heb geen aandelen Hemispherx.....

Ben slechts een patiŽnt die het middel graag zou proberen.




Hemispherx gets FDA denial on Ampligen treatment




December 3, 2009




Hemispherx Biopharma Inc. said Tuesday the Food and Drug Administration declined approval for the biotechnology company's experimental intravenous drug Ampligen as a treatment for chronic fatigue syndrome.









Hemispherx Biopharma Press Release for Tuesday, December 01, 2009



Hemispherx Biopharma Receives Complete Response Letter from FDA on Ampligen New Drug Application for Chronic Fatigue Syndrome



ÖOutlines Additional Recommendations



Philadelphia, PA, Tuesday, December 01, 2009: Hemispherx Biopharma, Inc. (NYSE Amex: HEB) (the "Company" or "Hemispherx"), announced that it recei≠ved a Complete Response Letter from the US Food and Drug Administration ("FDA") which describes specific additional recommendations related to the Ampligen NDA. In accordance with its 2008 "Complete Response" procedure, the FDA reviewers determined that they cannot approve the application in its present form and provided specific recommendations to address the outstanding issues. Hemispherx is carefully reviewing the Complete Response letter and will seek an expedited meeting with the FDA to discuss its recommendations. Management is pleased to have received specific advice on the remaining issues and is looking forward to making a thorough but expedited response its top priority, and plans to take all appropriate steps to seek approval and commercialization of Ampligen.


Most notably, the FDA stated that the two primary clinical studies submitted with the NDA did not provide credible evidence of efficacy of Ampligen and recommends at least one additional clinical study which shows a convincing effect and confirms safety in the target population. The FDA indicated that the additional study should be of sufficient size and sufficient duration (6 months) and include appropriate monitoring to rule out the generation of autoimmune disease. In addition, patients in the study should be on more than one dose regimen, including at least 300 patients on dose regimens intended for marketing. Finally, the additional study must incorporate both a well-controlled QT interval study and pharmacokinetic evaluations.


Other items required by the FDA include certain aspects of Non-Clinical safety assessment, and Product Quality. In the Non-Clinical area, the FDA is recommending that the Company complete rodent carcinogenicity studies in two species. As part of the NDA submission, the Company had requested that these studies be waived, but the waiver has not been granted. Certain additional non-clinical studies and additional data to support non-clinical studies already submitted with the NDA are also recommended by the FDA. Prior to the receipt of the Complete Response letter, the Company had already begun many of these additional studies and the collection of the requested additional data.


Under the Product Quality section of the Complete Response letter, the FDA recommends that the Company submit additional data and complete various analytical procedures. The collection of these data and the completion of these procedures is already part of the Companyís ongoing Quality Control, Quality Assurance program for Ampligen manufacturing under cGMP (current Good Manufacturing Practice Guidelines) and the manufacturing enhancement program recently undertaken by the Company and announced in a news release on September 16, 2009.


Finally, the FDA commented on Ampligen manufacturing noting the need to resolve outstanding inspection issues at the facilities producing Ampligen. These include the Company facility located in New Brunswick, NJ and one of the Companyís third party manufacturing facilities (Hollister-Stier Laboratories). The Company has been working to resolve these issues.


At this time the Companyís management has not determined the impact of the additional recommendations set forth in the Complete Response letter on the timelines and overall cost of the Ampligen program, but the Companyís management has made response to the issues and satisfaction of any additional requirements a top priority. The Company will seek to meet with the FDA to clarify any issues identified in the Complete Response letter and to work with the FDA to identify the most expeditious path to satisfaction of the requirements for approval of the Ampligen NDA.