Daniel Gueguen

Opened back in 2009, the “Orphacol file” is still not closed, despite three actions at the Court of Justice. What is to blame: the complexity of EU procedures or the Commission’s reluctance to recognise its errors of judgment? Probably a little of both!

It is extremely rare to go to the Court of Justice three times. Even more when it concerns a medicine used by 21 people – this is known as an “orphan medicine”. Here is a brief reminder of the story: in the early 90s a team of researchers developed a new active substance – cholic acid – capable of curing very young children suffering from liver deficiencies. Before, these children would have either died or undergone a liver transplant.
Two actions at the Court of Justice
In 2007, following 15 years of treatment, the Paris Hospitals authority decided to select a private company – CTRS Laboratoires – to develop, register and market the product under the name “Orphacol”. At the European Medicines Agency (EMA) in London, 28 Member States unanimously gave a green light to Orphacol’s authorisation…but the European Commission opposed it, invoking a number of legal and regulatory arguments that were ultimately found inaccurate. Submitted to the comitology procedure, the Member States twice opposed the Commission by qualified majority, effectively demanding the authorisation of Orphacol. But this didn’t work. CTRS launched a first action at the EU General Court for failure to act. During the proceedings, the Commission unilaterally decided to present its anti-Orphacol draft decision for a second time, submitting it to Member States on a public holiday (8 May 2012). As some Member States were absent, a qualified majority could not be reached. Orphacol had failed to obtain marketing authorisation.

The General Court was therefore obliged to declare CTRS’s action inadmissible. This led CTRS to file a new action, which turned out to be successful. On 25 October 2013, despite many twists and turns, notably the discovery of an American competitor called Asklepion Pharmaceuticals who clearly had the support of the DG SANTE services – the EU Official Journal published Orphacol’s marketing authorisation.
Then there was a third action
That should have been the end of the story, but it turned out that during this time, Asklepion had filed its own application for authorisation at the EMA for its own product Cholic Acid FGK. Even though CTRS now benefited from a 10-year market exclusivity under EU legislation on orphan medicines, the competitor’s procedure continued. Cholic Acid FGK received a positive EMA opinion, then an authorisation granted by the Commission via written procedure. Surprised by this decision, CTRS analysed Asklepion’s application in depth and discovered that Cholic Acid FGK had not been evaluated, not on its own merits, but with reference to Orphacol’s indications! Faced with this situation and lack of any dialogue with interested parties, CTRS turned to the Court for a third time. In June 2015, the judges confirmed the lack of an evaluation on the merits and annulled the marketing authorisation granted to Cholic Acid FGK (since then renamed “Kolbam”).
Court of Justice rulings continually disregarded
A happy ending? Not at all. Despite the latest decision of the General Court, the European Commission is still acting strangely on this file. On 7 September, bizarrely, DG SANTE asked the EMA to review its previous opinion on the efficacy and safety of Kolbam. On 24 September, barely two weeks later, the EMA adopted a “revised opinion” recommending that the Commission authorise Kolbam, completely in opposition to the Court ruling and the rules on commercial exclusivity for orphan medicines. What is the explanation for this new move? Nobody seems to know, and nobody at DG SANTE or in the upper levels of the Commission is responding to any demands for clarification.

At this stage, several questions must be asked: first, how can we accept such a waste of time, money and energy, involving three actions at the EU courts, for such a minor affair? And how could the EU authorities, in the wide sense of the word, go against the Court of Justice three times with such unparalleled arrogance and shamelessness? But this is also about the increased complexity of EU procedures leading to more and more errors, even though, in the present case, there is no question that DG SANTE – and by extension the whole Commission, given the media noise around the Orphacol file – still denies its mistakes and acts as if it was in the right all along! More than ever, it is essential that one day – or preferably as soon as possible – we find out the real reasons for this campaign against Orphacol and who instigated it.

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