Court overturns orphan refusal
pharmafile | July 10, 2013 | News story | Sales and Marketing | EMA, laboratoires ctrs, orphacol
A European Commission decision last year to refuse marketing authorisation for an orphan drug has been overturned by a major European court.
The General Court of Europe has annulled the EC ruling which did not allow privately-owned French company Laboratoires CTRS (Cell Therapies Research & Services) a licence for Orphacol.
The drug, which contains cholic acid, has been developed to treat rare but very serious – and potentially life-threatening – liver disorders.
The whole case throws light on some of the complications involved in getting an orphan drug to market – in particular the relative lack of clinical information that is available for regulators.
The main reason that the EC turned down Orphacol was that CTRS could not provide enough data to support its application.
CTRS applied to the European Medicines Agency on 30 October 2009, when only 90 patients had been diagnosed with the disorders, 19 of whom were treated in France.
The CHMP issued a positive opinion, followed by a revised opinion – also positive – in December 2010 and April 2011 respectively.
But in July 2011 the EC issued a draft decision refusing to grant CTRS a marketing authorisation and this was followed in January 2012 by CTRS bringing an action before the General Court.
However, the court found in effect that CTRS has done as much as it could by providing the regulator with bibliographical references to studies on cholic acid and “demonstrating that it was unable to provide comprehensive data for objective, verifiable reasons” – that is, that the disorders are so rare.
Also, since participation in a clinical trial would expose patients to the risk of serious liver damage – or even death – the EC was wrong to conclude that CTRS’s data should have been comprehensive.
The court made the point that cholic acid has been used to treat patients in France between 1993 and October 2007 in the form of hospital preparations provided on prescription under strict medical supervision.
Because these preparations were used to fulfil ‘special needs’ as defined by EU law – and because the active ingredient in Orphacol has been used for ten years in Europe effectively and safely – CTRS was under no obligation to provide the results of pre-clinical tests or clinical trials.
Thus the General Court has annulled the EC’s decision. CTRS could not be reached for comment.
Adam Hill
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