Brussels, 04/07/2013 (Agence Europe) - On Thursday 4 July, the General Court of the European Union annulled the European Commission's decision refusing to grant a marketing authorisation (MA) for Orphacol, a medicine developed by the Laboratoires CTRS for the treatment of severe, albeit rare, liver disorders. The Court ruled that the orphan medicine could be marketed on the basis of well-established medicinal use dating back more than ten years.
In its decision of 25 May 2012, the Commission refused to give a favourable answer to the CTRS' application, in October 2009, to the European Medicines Agency (EMA) for MA of the medicinal product. This was despite the various positive opinions regarding marketing of the product, first of all from the Standing Committee on Medicinal Products for Human Use in 2010 and 2011, and then from the Appeal Committee in November 2011. In order to justify its decision, the Commission invoked the lack of documents from CTRS to show that there was well-established medicinal use of the product in question, and said there was no evidence of it being safe or effective.
The General Court refused these arguments and annulled the Commission's decision, noting that: - cholic acid, the active substance in Orphacol, was used to treat patients in France between 1993 and October 2007 in the form of hospital preparations provided on medical prescription, prepared individually in accordance with the prescriptions of a pharmacopoeia and in compliance with the rules of good practice; - since then, cholic acid capsules have been authorised for use in France under the brand name Orphacol; - the Court found that those hospital preparations were intended to fulfil “special needs” as defined in EU law on human medicines (Directive 2001/2001/83/EC), i.e. in individual situations justified by medical considerations and necessary to meet patients' needs. Under these circumstances, the Court states, CTRS was not under an obligation, when faced by the request for MA, to provide the results of non-clinical and clinical summaries required by the directive, as the Commission had requested. Furthermore, CTRS showed that it was not able to provide complete information on the effectiveness and safety of the medicine under normal circumstances due to the exceptional circumstances recognised by EU law (rareness of the ailment, risks for patients taking part in clinical trials given their exposure to severe liver damage).
Socialist MEP Gilles Pargneaux of France welcomed the ruling, and denounced the Commission's “bureaucratic stubbornness” in this affair. He said: “Some manoeuvres, including the fact of convening as many times as it wanted the member state committee of examination until it got what it wanted, remains to be elucidated. This refusal to grant MA was unprecedented. Usually the European Commission systematically follows the opinion of the EMA which, from the start, had recognised that the medicinal product was vital. Justice has been done”. (FG/transl.jl)