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Court orders EC to approve rejected orphan medicine

Says CTRS should be allowed to market Orphacol for the treatment of rare bile condition
European Court of Justice

The European Court of Justice (ECJ) has instructed the European Commission (EC) to approve an orphan medicinal product that it had previously rejected for marketing, in the latest chapter of a three-year legal dispute.

The battle pitched small French pharma company Cell Therapies Research & Services (CTRS) against the Commission and focused on Orphacol (cholic acid), a drug intended for the treatment of inborn errors in primary bile acid synthesis.

These serious but rare conditions lead to liver dysfunction that if not properly treated can lead to death within the first weeks or months after birth.

The EC's decision to refuse approval to Orphacol in May 2012 came despite a recommendation from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) that it should be licensed under 'exceptional circumstances' as its benefits were greater than its risks.

The decision to turn down the application was made despite opposition from EU national authorities and stemmed from the fact that CTRS had provided mainly data from the scientific literature in support of the application, attempting to get approval on the basis of well-established medicinal use dating back more than 10 years.

To make use of that regulatory pathway the company had to prove that it was unable to carry out the usual testing of a new medicine because the diseases are vanishingly rare and there are ethical reasons not to test the treatment concept clinically.

"The Court notes that cholic acid has been used to treat patients in France between 1993 and October 2007 in the form of hospital preparations provided on medical prescription," said the ECJ's General Court in a statement.

"Thus CTRS was not obliged, when applying for the [market authorisation], to provide the results of pre-clinical tests or clinical trials required by EU law," it added. The EC has leave to appeal the decision, provided it responds to the General Court decision within two months.

The case has been held up by some as evidence that the entire EU medicines registration system is flawed.

An article in EuroPolitics by Daniel Guéguen visiting professor at the College of Europe, argues that the Commission's decision-making process is becoming overly bureaucratic and beset by competing interests, both within the directorates-general and at the national level.

Its power to adopt regulatory texts is also almost unchallenged, claims Guéguen, as the counter-power held by the European Parliament and Council "is considerably weakened, often even non-existent or not operational".

11th July 2013

From: Sales, Regulatory



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