|HEB Gets Response Letter From FDA|
|By Staff and Wire Reports|
|Tuesday, 01 December 2009 17:12|
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 Company's 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 Company's third party manufacturing facilities (Hollister-Stier Laboratories). The Company has been working to resolve these issues.
At this time the Company's 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 Company's management has made response to the issues and satisfaction of any additional requirements a top priority.