Pharma World

Glybera marketing authorisation application in Limbo (January 31, 2012)

Published with kind permission of MEDNOUS
In an apparently unprecedented situation, a committee of the European Union member states which advises the European Commission on the granting or refusal of marketing authorisations, has reached an impasse over the gene therapy, Glybera.

According to the therapy’s sponsor, Amsterdam Molecular Therapeutics BV, the committee (Standing Committee on Medicinal Products for Human Use) has asked for more information before taking a position.


Under European pharmaceutical regulation procedures, the European Medicines Agency, and specifically its Committee for Medicinal Products for Human Use (CHMP), recommends the approval, or the denial, of applications from companies which seek to market their new drugs. The opinions of the CHMP are nearly always endorsed by the Standing Committee, which advises the European Commission. The Commission then issues, or denies, a marketing authorisation.


Now, the Standing Committee has apparently reached an impasse over Glybera (alipogene tiparvovec), a gene therapy for a rare genetic disorder. Glybera was turned down by the CHMP in June 2011 and then again on appeal, in October on efficacy grounds. The therapy is intended to treat a deficiency of the lipoprotein lipase protein, a condition that can lead to pancreatitis.


At a meeting of the Standing Committee on 22 January, 2012 however “no clear position in favour or against granting a marketing authorisation for Glybera was reached,” Amsterdam Molecular Therapeutics said in a press release issued on 30 January. “Instead the Standing Committee considered it necessary to request additional information from the CHMP…A formal vote by the Standing Committee will be made on review of the additional information. It is currently unclear when a final decision will be reached,” the company said.


On 20 October 2011, the CHMP turned down an appeal from Amsterdam Molecular Therapeutics which sought to have a previous negative opinion – recommending refusal of a license – overturned. In turning down the appeal, the CHMP overruled another specialist committee, the Committee for Advanced Therapies, which thought the drug should be let onto the market, albeit with close monitoring. The Standing Committee, which usually rubber-stamps CHMP opinions, reportedly hasn’t yet been able to take a decision either way.

The Glybera case appears to have raised new issues for the regulators about the standards and procedures that should apply in approving novel medicines such as gene therapies. To date, no gene therapy has ever been approved in Europe or in the US.


Copyright 2012 Evernow Publishing Ltd


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