The European Court of Justice (ECJ) has thrown out a decision by the European Commission (EC) not to approve Orphacol, an orphan drug manufactured by French pharma company Cell Therapies Research and Services (CTRS). Orphacol is intended for the treatment of rare inborn errors in primary bile acid synthesis, and its active ingredient cholic acid has been used in French hospitals since 1993.
The EMA’s Committee for Medicinal Products for Human Use (CMPHU) originally recommended that the drug should be granted an MA. Because the liver indication is so rare, CTRS had instead submitted references to scientific literature to back up its application rather than the results of clinical trials – fulfilling the criteria for demonstrating “well-established medicinal use”. However, the European Commission refused to grant an MA for the drug, saying that the French hospital preparation use in patients since the early 1990s did not fulfil the criteria for demonstrating well-established medicinal use.
The European Court has now ruled that the well-established medicinal use had been demonstrated by CTRS, and accepts that the rareness of the disorders and the risk of serious liver damage associated with a clinical trial means that CTRS can not carry out a controlled study of the drug’s efficacy.
“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,” the Court said.
The case is sure to ignite discussion on the complications of regulating orphan drugs, especially in cases where there is a relative lack of clinical information.
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