statistical significance due to low study power shows despite clinically relevant large effect sizes in the paediatric subpopulation. Advice from the ACPM is requested. Proposed action Given the above findings and my comments, the Delegate is of the view that the current submission has demonstrable deficiencies in all areas that is, Quality, Toxicology and – Clinical and thus its efficacy and safety were not satisfactorily shown based on the data – in the submitted dossier. The Delegate accepts the drug will be used in highly specialised units by highly trained physicians. However, this in itself is not an alternative to reliable evidence of effectiveness for general marketing authorisation. Please also note that remestemcel-L (ex vivo hMSCs), as product derived from blood, is an unscheduled substance under the Poisons Schedule, that is, it has not been classified as prescription only medicine. However, given the expected use of this drug only in specialised circumstances this is not considered an issue. Please also note that such submissions now fall the new Biologicals regulatory framework whereby an orphan designation is no longer available which may be relevant to any future submission for this product. The Delegate thanks the Committee for considering this submission and providing advice to the TGA. Outcome On 18 November 2012, Delpharm Consultants Pty Limited wrote to the TGA, asking for the application for Prochymal to be withdrawn before it was presented for consideration by the Advisory Committee on Prescription Medicines. Attachment 1. Extract from the Clinical Evaluation Report
statistical significance due to low study power shows despite clinically relevant large effect sizes in the paediatric subpopulation. Advice from the ACPM is requested. Proposed action Given the above findings and my comments, the Delegate is of the view that the current submission has demonstrable deficiencies in all areas – that is, Quality, Toxicology and Clinical – and thus its efficacy and safety were not satisfactorily shown based on the data in the submitted dossier. The Delegate accepts the drug will be used in highly specialised units by highly trained physicians. However, this in itself is not an alternative to reliable evidence of effectiveness for general marketing authorisation. Please also note that remestemcel-L (ex vivo hMSCs), as product derived from blood, is an unscheduled substance under the Poisons Schedule, that is, it has not been classified as prescription only medicine. However, given the expected use of this drug only in specialised circumstances this is not considered an issue. Please also note that such submissions now fall the new Biologicals regulatory framework whereby an orphan designation is no longer available which may be relevant to any future submission for this product. The Delegate thanks the Committee for considering this submission and providing advice to the TGA. Outcome On 18 November 2012, Delpharm Consultants Pty Limited wrote to the TGA, asking for the application for Prochymal to be withdrawn before it was presented for consideration by the Advisory Committee on Prescription Medicines. Attachment 1. Extract from the Clinical Evaluation Report
auspar-remestemcel-l-150315.docx