The General Court of the European Union (EU) has annulled a decision by the European Commission to refuse a marketing authorisation (MA) for an orphan drug.
The case concerns Laboratoires CTRS' Orphacol (cholic acid), which is used in the treatment of two rare but very serious liver disorders. In October 2009, CTRS applied to the European Medicines Agency (EMA) for an MA for Orphacol. In December 2010, the EMA's Committee for Medicinal Products for Human Use (CMPHU) issued a positive opinion on the drug, and in April 2011 it issued a revised opinion, also positive, recommending that it be granted an MA.
However, in October 2011 the Commission submitted a draft decision to the Standing Committee on Medicinal Products for Human Use refusing to grant CTRS an MA for Orphacol, on the grounds that it had not followed the legal requirements for obtaining an MA. Then in November 2011, it sent the same draft decision to the Appeal Committee. Both the Standing Committee and the Appeal Committee rejected the Commission's draft decision.
CTRS then brought the case before the EU General Court but, on the eve of the hearing, "the Commission derailed the Court procedure by replacing its original proposal with a different version - simply re-worded and still refusing the MA," says law firm Bristows LLP, which represents CTRS in this case.
On May 25, 2012, the Commission issued another decision refusing to grant an MA to Orphacol, and on July 10, CTRS again brought action before the General Court seeking annulment of this latest refusal.
The Court decided to give the case priority treatment, and the judgment given this this month it has annulled the Commission's decision of May 25, 2012.
In its ruling, the General Court says that CTRS was not obliged, when applying for the MA, to provide the results of pre-clinical tests or clinical trials required by EU law, because cholic acid had been used in French hospitals since 1993 "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 laid down in French legislation." These hospital preparations had also had been supplied to fulfil "special needs" defined in EU law.
The Court therefore rejected the Commission's arguments that there was no well-established medicinal use - this had in fact been demonstrated by CTRS.
It also finds that CTRS had shown it was unable to provide "comprehensive particulars" on Orphacol’s safety and efficacy under normal conditions of use for "objective, verifiable reasons." The first of these was the rareness of the disorders in question - when the MA applications was submitted, on October 30, 2009, only 90 patients had been diagnosed with the disorders, 19 of whom were treated in France.
Secondly, because participation in a clinical trial would expose patients to the risk of serious liver damage, or even death, it would be contrary to the principles of medical ethics to carry out a controlled study of the drug's efficacy, says the Court.
"Accordingly, the Commission was wrong to conclude in its decision that the data submitted by CTRS should have been comprehensive, and that it could not invoke the existence of exceptional circumstances in its application made on the basis of well-established medicinal use," added the Court.
The decision was welcomed as "a landmark ruling, not just for the industry but for EU citizens as a whole" by Marie Manley, partner and head of the regulatory practice at Bristows, who said: "it leaves no doubt on how incorrect the Commission was to hide behind its status as 'guardian of the treaties' in refusing to authorise Orphacol."
"The Court has pulled the rug from under the Commission's feet by ruling that the Commission had no legal basis for refusing the MA. This judgement is the first to provide guidance on what type of use qualifies under the 'well-established use' procedure for obtaining an MA, and the first to interpret the 'exceptional circumstances' provision," she said.
It was unfortunate, Ms Manley added, that the Court felt it did not need to rule on the "comitology" aspects of the case - the rules which set out the balance of powers between EU member states and the Commission - as this will leave stakeholders in the many industries where decisions are subject to the comitology procedure in a limbo.
However, she added: "I expect that the Commission will be humbled by the Court's ruling and will now take the appropriate steps to comply with the Court's judgement, namely by authorising Orphacol without further delay."